On March 3, 2026 AN2 Therapeutics, Inc. (Nasdaq: ANTX), a clinical-stage biopharmaceutical company developing novel small molecule therapeutics derived from its boron chemistry platform, reported its plans to expand the development of oral epetraborole into a Phase 2 proof-of-concept clinical study in adults with phlebotomy-dependent polycythemia vera (PV). PV is a blood cancer characterized by overproduction of red blood cells in the bone marrow. This overproduction increases hematocrit which can lead to serious medical complications, including arterial and venous thromboembolic events.

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The Company’s decision to pursue PV is supported by data from multiple clinical trials of oral epetraborole in healthy volunteers and non-PV patients in which the drug consistently demonstrated early, controlled, sustained, and dose-dependent reductions in hematocrit at potentially clinically meaningful levels for PV.

Epetraborole’s effects were characterized by the following pharmacodynamic and clinical observations:

Consistent hematocrit reductions across multiple clinical populations, including healthy volunteers and nontuberculous mycobacterial (NTM) lung disease patients, with effects sustained over a six-month treatment period
Early onset of hematocrit reduction after dose initiation with durable, stable control and reversibility at treatment cessation
No clinically relevant change in white blood cell counts and minimal change in platelet counts
Demonstrated durable hematocrit reduction in 9-month chronic non-human primate studies
A potentially differentiated mechanism of action, likely acting on globin synthesis rather than directly on heme synthesis
Epetraborole has been generally well tolerated in clinical trials to date at doses anticipated for the treatment of PV.

"We believe epetraborole may offer a differentiated hematological profile that combines hematocrit control via red-cell selectivity, early onset, titratability and oral delivery, attributes that could address key treatment objectives in polycythemia vera and offer patients a new therapeutic option where current approaches fall short," said Eric Easom, Co-Founder, Chairman, President and CEO of AN2 Therapeutics. "This program creates additional, near-term value inflection points within our current runway and broadens our pipeline, which now includes three Phase 2 studies initiating in 2026, and two preclinical oncology compounds that are expected to move into development this year."

"While current therapies are effective for some patients, many continue to have inadequately controlled hematocrit levels and rely on repeated phlebotomy or injectable treatments," said Stan Gerson, M.D., Hematologist and Oncologist at University Hospitals Cleveland Medical Center and Dean and Professor of Medicine at Case Western Reserve University School of Medicine. "As a chronic illness with no cure, PV carries a persistent risk of thrombosis and a substantial symptom burden. There remains a clear need for additional oral treatment options, including those with novel mechanisms of action, that can help manage hematocrit while minimizing treatment burden and long-term tolerability concerns."

The Company is currently proceeding through the regulatory clearance process and anticipates initiating the Phase 2 trial in the third quarter of 2026. The Company expects to provide periodic public data updates as early as the fourth quarter of 2026, subject to regulatory clearance and enrollment progress.

About Polycythemia Vera (PV)

PV is a blood cancer characterized by overproduction of red blood cells in the bone marrow. This overproduction increases hematocrit which can lead to serious medical complications, including arterial and venous thromboembolic events. If untreated, PV can be life-threatening. Despite available therapies, many patients experience uncontrolled hematocrit levels and persistent symptom burden, requiring long term management to maintain adequate disease control. PV is estimated to affect approximately 155,000 people in the U.S.

Webcast Information

AN2 will host a live webcast presentation on Wednesday, March 4, 2026 at 9:30am ET to provide an overview of the PV program. The event will feature Dr. Aaron Gerds, alongside members of the AN2 team. Dr. Gerds is the Associate Professor of Medicine at the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University and serves as Deputy Director for Clinical Research at the Cleveland Clinic Taussig Cancer Institute. He is also Deputy Associate Director for Clinical Research at the Case Comprehensive Cancer Center.

The live webcast of the presentation can be accessed by registering under "Events" in the investors section of the Company’s website at View Source or View Source Upon registration, all participants will receive an email confirmation with a link that will log you in automatically and the option to add it to your calendar. It is recommended that participants log into the webcast approximately 10 minutes prior to the webcast. An archived replay will be available for 30 days following the presentation. The replay will be available on this same link beginning approximately two hours after the event.

About the Phase 2 Study of Epetraborole in PV

This planned Phase 2 study consists of an open-label epetraborole sentinel cohort, an open-label dose optimization cohort for dose selection (Part 1), followed by a double-blind, randomized, placebo-controlled cohort (Part 2), and an optional open-label extension cohort (Part 3). The study is designed to assess the efficacy of oral epetraborole in phlebotomy-dependent adults with PV and its effect on key hematological variables, to optimize a dose regimen on a by-patient level, to assess safety and tolerability, to assess patient-reported outcomes (PROs) using validated PRO instruments, and to assess other key hematological parameters.

About Epetraborole

Epetraborole is a boron-containing, orally available, small molecule that has shown dose and exposure-dependent decreases in hematocrit. Evidence suggests that it operates by reducing production of early-stage erythrocytes (red blood cells) while sparing other cell lineages in the marrow, including white blood cells and platelets. Epetraborole’s clinical data package supporting evaluation in PV is comprehensive, including 10 Phase 1 studies, two Phase 2 studies, and a Phase 2/3 study in NTM lung disease. The drug has been generally well tolerated in prior trials at doses anticipated for PV and, to date, no tolerability barriers to long-term use have been identified. Epetraborole, if approved, would represent a distinct chemical class in both PV and anti-infectives.

(Press release, AN2 Therapeutics, MAR 3, 2026, View Source [SID1234663268])

On March 3, 2026 Propanc Biopharma, Inc. (Nasdaq: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company focused on developing novel treatments for chronic diseases, including recurrent and metastatic cancer, reported the potential of its lead asset, PRP, as a novel therapeutic approach to the treatment and prevention of metastatic cancer from solid tumors, especially more aggressively spreading, less differentiated tumors, which offer a poor patient prognosis. Pancreatic cancer is one of the deadliest cancers, with a five-year survival rate stuck at just 13% and no real progress has been made in recent years. To put that into perspective, overall cancer survival is 70%.

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Standard treatments like chemotherapy (FOLFIRINOX or gemcitabine/nab-paclitaxel), targeted therapies (e.g., KRAS inhibitors), and emerging options (immunotherapies, tumor-treating fields like Optune Pax) extend life modestly but often bring harsh side effects, resistance, and limited success against this aggressive, metastasis-prone disease.

Enter Propanc’s PRP—a promising investigational proenzyme therapy (trypsinogen + chymotrypsinogen in a 1:6 ratio) delivered intravenously. Unlike cytotoxic drugs that kill dividing cells broadly, PRP targets cancer stem cells, blocks metastasis by suppressing epithelial-mesenchymal transition (EMT), disrupts the tumor microenvironment, curbs angiogenesis, and boosts chemosensitivity—potentially making standard treatments more effective with far less toxicity.

Preclinical data shines: >85% tumor growth inhibition in pancreatic models, reduced fibrosis and resistance markers, and a gentler profile (no major side effects in limited prior human use). A small compassionate study (rectal version) extended survival from ~5.6 to 9 months in advanced cases.

PRP vs. Current Treatment Options:

Chemo: PRP could sensitize resistant tumors and cut doses/side effects.
Targeted drugs: Broader attack on stem cells and spread, not just single mutations.
Immunotherapy: May warm up "cold" pancreatic tumors by remodeling the microenvironment.
According to industry sources the global pancreatic cancer treatment market is valued at ~$4.42 billion in 2026 and projected to explode to $14.43 billion by 2034 (CAGR ~16%), fueled by rising cases and demand for better options.

Propanc is gearing up for a Phase 1b First-In-Human trial in 2026 (30–40 advanced solid tumor patients), backed by fresh funding ($100M facility), new patents, and FDA Orphan Drug status for pancreatic cancer.

"We are excited about PRP’s potential to transform cancer care by targeting the underlying mechanisms of metastasis with a mechanism that could offer meaningful advantages over existing therapies," said James Nathanielsz, Propanc’s Chief Executive Officer. "PRP remains experimental—no large human efficacy data yet—but its multi-targeted, low-toxicity approach could redefine care for a disease desperate for breakthroughs," Mr. Nathanielsz concludes.

(Press release, Propanc, MAR 3, 2026, View Source [SID1234663229])

On March 3, 2026 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine, and Merck, known as MSD outside of the United States and Canada, reported an expanded, multi-year collaboration aimed at accelerating the discovery and development of precision medicine biomarkers and supporting Merck’s oncology and potentially broader therapeutic portfolios.

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"This collaboration builds on our existing relationship and reflects our shared commitment to harnessing the power of multimodal datasets with AI to deliver better options for patients," said Ryan Fukushima, CEO, Data & Apps at Tempus. "We’ve spent years configuring our Lens Platform to seamlessly leverage our library of de-identified multimodal data with the necessary AI computing power to train and fine-tune specific models for healthcare. Working with the great scientists at Merck, we have exciting opportunities to translate the insights from AI models into precision medicine strategies and improve patient outcomes across oncology and beyond."

Under the terms of the agreement, Merck will use Tempus’ de-identified data along with Tempus’ Lens Platform and Workspaces environment, which offers an advanced computational configuration powered by one of the industry’s largest GPU infrastructures, which enables researchers to efficiently conduct complex analyses on training-ready multimodal datasets, generating novel insights to accelerate the development and optimization of candidate therapies at scale.

"The combination of new AI technologies and large curated multimodal data sets are transforming the way we conduct discovery research," said George Addona, Senior Vice President, Discovery, Preclinical Development and Translational Medicine, Merck Research Laboratories. "This collaboration with Tempus positions Merck to advance our precision oncology strategy through the application of the latest AI/ML capabilities to discover novel precision biomarkers, identify mechanisms of cancer cell resistance, and inform rational combinations for drugs in our early pipeline."

(Press release, Tempus, MAR 3, 2026, View Source [SID1234663269])

On March 3, 2026 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today reported financial results for the fourth quarter and year ended December 31, 2025, including sales of TAVALISSE (fostamatinib disodium hexahydrate), GAVRETO (pralsetinib) and REZLIDHIA (olutasidenib), and recent business progress.

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"I am proud to highlight Rigel’s tremendous progress during 2025 across each of the key value drivers of our business. We delivered record net product sales, total revenues and net income while making meaningful advances in our Phase 1b study of R289 in lower-risk MDS," said Raul Rodriguez, Rigel’s president and CEO. "These 2025 accomplishments set the stage for a strong 2026, as reflected in our financial guidance and our plans to advance our R289 program in lower-risk MDS and other potential indications."

Fourth Quarter and Full Year 2025 Business Update

Commercial

Fourth quarter net product sales were $65.4 million, an increase of 41% from the same period of 2024.
2025 net product sales were $232.0 million, an increase of 60% from the same period of 2024.
Clinical Development and Regulatory

Rigel continues to advance its Phase 1b clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R2891, a potent and selective dual inhibitor of interleukin receptor-associated kinases 1 and 4 (IRAK1/4), in patients with relapsed or refractory (R/R) lower-risk myelodysplastic syndrome (MDS). In October 2025, Rigel announced enrollment of the first patient in the dose expansion phase of the study, where up to 40 patients will be randomized to receive either 500 mg once daily (QD) or twice daily (BID) to determine the recommended Phase 2 dose for future clinical trials.
Rigel is on track to complete enrollment of the dose expansion phase of the Phase 1b study and select the recommended Phase 2 dose for future clinical studies in the second half of 2026. The company anticipates sharing preliminary data from the dose expansion phase of the study by the end of 2026.
Updated data from the dose escalation phase of the ongoing Phase 1b clinical study of R289 were presented in an oral session at the 2025 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December, indicating that R289 continued to be generally well tolerated in a heavily pretreated R/R lower-risk MDS patient population, the majority of whom were high transfusion burden (HTB) at baseline. Furthermore, red blood cell transfusion independence (RBC-TI ≥8 weeks) was achieved by 33% (6/18) of evaluable transfusion dependent patients receiving R289 doses of 500 mg QD and higher.
Also at the ASH (Free ASH Whitepaper) Annual Meeting, four posters were presented on olutasidenib, which included data that add to the growing body of evidence supporting the benefits of its use in patients with R/R mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML).
In October 2025, the first patient was enrolled in the CONNECT Phase 2 TarGeT-D study evaluating olutasidenib in combination with temozolomide, followed by olutasidenib monotherapy as a maintenance regimen for newly-diagnosed adolescent and young adult patients with a high-grade glioma harboring an IDH1 mutation (NCT06161974).
Rigel presented sub-analysis data from the ARROW study evaluating pralsetinib for the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) at the 2025 North America Conference on Lung Cancer (NACLC) in December. The two poster presentations included efficacy and safety data for patients previously treated with immunotherapy and for patients from the United States. In the trial, median overall survival (OS) was 44.3 months for the overall patient population. Longer median OS was seen in patients treated in the United States (62.4 months).
Rigel presented the first data release for pralsetinib from the TAPISTRY study (NCT04589845) in a poster presentation at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) – Gastrointestinal Cancers Symposium (ASCO-GI) in January. The analysis reported results from the Phase 2 global, open-label, multicohort study, in which the efficacy and safety of pralsetinib was evaluated in a cohort of patients with RET fusion-positive solid tumors, including pancreatic, colorectal, and hepatobiliary cancers. Pralsetinib demonstrated robust and durable activity against RET fusion-positive solid tumors, including gastrointestinal (GI) tumors, and in the efficacy evaluable population showed an overall response rate (ORR) of 67% (26/39). These data validate RET fusions as a tissue-agnostic target with sensitivity to RET inhibition, suggesting the potential therapeutic utility of pralsetinib in these patients.
On December 22, 2025, the U.S. Food and Drug Administration (FDA) notified Rigel of the approval of a Prior Approval supplemental New Drug Application for GAVRETO, which updated the U.S. Prescribing Information to add a boxed warning regarding serious infections, including opportunistic infections. Rigel previously communicated this risk information to healthcare providers via a Dear Healthcare Provider letter in October 2024. The FDA also notified Rigel that it has met its postmarketing commitment for GAVRETO from its September 2020 accelerated approval to submit the final report for the AcceleRET-Lung study. In October 2023, the FDA granted full approval to GAVRETO for adult patients with metastatic RET fusion-positive NSCLC.
Publication

A paper titled "Olutasidenib for Mutated IDH1 Acute Myeloid Leukemia: Final Five-Year Results from the Phase 2 Pivotal Cohort" was published in November 2025 by Jorge E. Cortes, M.D., Phase 2 trial investigator and Director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, in the Journal of Hematology & Oncology. The publication reports the final 5-year data from the pivotal cohort of the registrational trial evaluating olutasidenib for the treatment of patients with R/R mIDH1 AML, which includes an additional two years of efficacy and safety data. These 5-year data further support the durable responses and manageable safety profile observed with olutasidenib in patients with R/R mIDH1 AML, including those previously treated with venetoclax-based regimens.
Corporate

Michael P. Miller joined Rigel’s Board of Directors as an independent director and member of the Board of Directors’ Compensation Committee, effective February 1, 2026.
Fourth Quarter and Full Year 2025 Financial Update

For the fourth quarter ended December 31, 2025, total revenues were $69.8 million, consisting of $65.4 million in net product sales and $4.4 million in contract revenues. Net product sales grew 41% compared to $46.5 million in the same period of 2024. TAVALISSE net product sales were $45.6 million, growth of 47% compared to $31.0 million in the same period of 2024. GAVRETO net product sales were $10.2 million, growth of 27% compared to $8.1 million in the same period of 2024. REZLIDHIA net product sales were $9.6 million, growth of 29% compared to $7.4 million in the same period of 2024. Contract revenues include $4.1 million in contract revenues from collaborations and $0.3 million in government contract revenue. Contract revenues from collaborations primarily consisted of $3.4 million of revenue from Grifols S.A. (Grifols) related to delivery of drug supplies and earned royalties, $0.3 million of revenue from Kissei Pharmaceutical Co., Ltd. (Kissei) related to the delivery of drug supplies and $0.2 million of revenue from Medison Pharma (Medison) related to earned royalties.

Total costs and expenses were $46.6 million compared to $40.9 million for the same period of 2024. The increase in costs and expenses was mainly due to increased research and development costs driven by the timing of clinical activities related to R289 and olutasidenib and higher personnel-related costs.

Income before income taxes was $22.7 million. Benefit from income taxes was $245.4 million in the fourth quarter, primarily driven by $245.9 million of non-cash deferred income tax benefit, partially offset by state tax expenses.

Rigel reported net income of $268.1 million, or $14.72 basic and $13.54 diluted per share, compared to $14.3 million, or $0.81 basic and $0.80 diluted per share, for the same period of 2024.

For the year ended December 31, 2025, total revenues were $294.3 million, consisting of $232.0 million in net product sales and $62.3 million in contract revenues. Net product sales grew 60% compared to $144.9 million in the same period of 2024. TAVALISSE net product sales were $158.8 million, growth of 52% compared to $104.8 million in the same period of 2024. GAVRETO net product sales were $42.1 million, growth of 146% compared to $17.1 million in the same period of 2024. GAVRETO became commercially available from Rigel in late June 2024. REZLIDHIA net product sales were $31.0 million, growth of 35% compared to $23.0 million in the same period of 2024. Contract revenues include $62.0 million in contract revenues from collaborations and $0.3 million in government contract revenue. Contract revenues from collaborations primarily consisted of $40.0 million in non-cash revenue resulting from the release of the remaining cost share liability related to the agreement with Lilly for the development and commercialization of ocadusertib, $13.2 million of revenue from Grifols related to delivery of drug supplies and earned royalties, $7.2 million of revenue from Kissei related to a milestone payment and delivery of drug supplies and $1.1 million of revenue from Medison related to delivery of drug supplies and earned royalties.

Total costs and expenses were $168.8 million compared to $155.1 million for the same period of 2024. The increase in costs and expenses was mainly due to increased research and development costs driven by the timing of clinical activities related to R289 and olutasidenib, higher personnel-related costs, and higher cost of product sales.

Income before income taxes was $121.8 million. Benefit from income taxes was $245.2 million in 2025, primarily driven by $245.9 million of non-cash deferred income tax benefit, partially offset by state tax expenses.

Rigel reported net income of $367.0 million, or $20.40 basic and $19.48 diluted per share, compared to $17.5 million, or $0.99 basic and diluted per share, for the same period of 2024.

Cash, cash equivalents and short-term investments as of December 31, 2025 was $155.0 million, compared to $77.3 million as of December 31, 2024.

2026 Outlook
Rigel anticipates 2026 total revenues of approximately $275 to $290 million, including:

Net product sales of approximately $255 to $265 million.
Contract revenues of approximately $20 to $25 million.
The company anticipates it will report positive net income for the full year 2026, while funding existing and new clinical development programs.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time
Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About ITP
In patients with immune thrombocytopenia (ITP), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. Patients suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About NSCLC
It is estimated that over 229,000 adults in the U.S. will be diagnosed with lung cancer in 2026. Lung cancer is the leading cause of cancer death in the U.S., with non-small cell lung cancer (NSCLC) being the most common type accounting for 77% of all lung cancer diagnoses.2 RET fusions are implicated in approximately 1-2% of patients with NSCLC.3

About AML
Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that there will be about 22,720 new cases in the United States, most in adults, in 2026.4

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.5,6 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.7 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need.

About TAVALISSE
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Please click here for Important Safety Information and Full Prescribing Information for TAVALISSE.

About GAVRETO
GAVRETO is indicated for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).*

*Thyroid indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

Please click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING, for GAVRETO.

About REZLIDHIA
REZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

Please click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING, for REZLIDHIA.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

TAVALISSE, GAVRETO and REZLIDHIA are registered trademarks of Rigel Pharmaceuticals, Inc.

(Press release, Rigel, MAR 3, 2026, View Source [SID1234663230])

On March 3, 2026 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the fourth quarter and year ended December 31, 2025.

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"Meaningful scientific and operational progress in 2025 has positioned us well to generate human proof-of-concept data over the next 12-18 months for SC451 in type 1 diabetes and SG293 in blood cancers," said Steve Harr, Sana’s President and Chief Executive Officer. "Clinical data for UP421, a study which is now out beyond a year, provide the first known example of transplanting an allogeneic cell therapy for the treatment of type 1 diabetes without any immunosuppression. These results, when combined with progress in the field of transplanting pancreatic islets, make us optimistic that SC451, which incorporates the same hypoimmune gene edits into a more scalable manufacturing platform, can lead to a functional cure for people with type 1 diabetes, meaning normal blood glucose, no more insulin injections, and no immunosuppression. Moving to the fusogen platform, we made improvements to our in vivo CAR T platform with our next‑generation SG293 candidate, offering the potential for a simple, one-time, off-the-shelf treatment without the use of conditioning chemotherapy for the treatment of B cell cancers and B cell-mediated autoimmune diseases. We look forward to beginning clinical trials for both of these therapies this year. With two powerful platforms advancing in parallel, we look to drive meaningful clinical benefit for patients."

Corporate Highlights

Published positive results from an investigator-sponsored, first-in-human study transplanting UP421, an allogeneic primary islet cell therapy engineered with hypoimmune platform (HIP) technology, into a patient with type 1 diabetes without the use of any immunosuppression.

UP421 is a primary human HIP-modified pancreatic islet cell therapy for patients with type 1 diabetes. The goal of this investigator-sponsored trial (IST) was to understand safety, immune evasion, islet cell survival, and beta cell function, as measured by C-peptide production, of HIP-modified pancreatic islet cells transplanted into a type 1 diabetes patient without the use of any immunosuppression. The trial is being conducted under a clinical trial authorization at Uppsala University Hospital with Dr. Per-Ola Carlsson as the principal investigator.
Results of the study through one year after cell transplantation demonstrate the survival and function of pancreatic beta cells as measured by the presence of circulating C-peptide, a biomarker indicating that transplanted beta cells are producing insulin. C-peptide levels also increase with mixed meal tolerance tests performed over the course of the study, consistent with insulin secretion in response to a meal. PET-MRI scanning performed at week 12 and again at week 52 demonstrated islet cells at the transplant site in the forearm. The study identified no safety issues, and the HIP-modified islet cells evaded immune detection.
The New England Journal of Medicine (NEJM) published a journal article titled "Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression" (DOI: 10.1056/NEJMoa2503822). The article discusses 12-week results from this study. NEJM also published an accompanying editorial that further describes both the Sana technology and progress in the field (DOI: 10.1056/NEJMe2507578).

Advancing our focused pipeline across two platforms:

Hypoimmune Platform – Type 1 diabetes – Sana continues development of SC451, an O-negative, HIP-modified, iPSC-derived pancreatic islet cell therapy, which uses the same HIP technology as UP421. Sana has had multiple interactions with regulators over the last year, including FDA INTERACT and Pre-IND meetings, the results of which increase confidence in its manufacturing process, manufacturing controls, nonclinical testing plan, and clinical trial plan. Sana expects to file an IND and begin a Phase 1 clinical trial for SC451 as early as this year.
Fusogen Platform – In vivo CAR T cells – Sana continues to develop its next-generation in vivo CAR T product candidate, SG293, which uses Sana’s proprietary fusogen delivery-based technology. SG293 is a CD8-targeted fusosome that delivers to CD8+ T cells the genetic material to make CD19-directed CAR T cells while avoiding potentially troublesome delivery to tissues such as the liver. Preclinical data demonstrate that a SG293 surrogate achieves cell-specific delivery and deep B-cell depletion – as measured by depletion in circulating and lymph node B cells as well as a phenotypic reset when B cells return – in non-human primates without the use of any lymphodepleting chemotherapy. Sana intends to explore SG293 in both B-cell cancers and B-cell mediated autoimmune diseases and expects to generate first-in-human data as early as this year.

Published preclinical data in Nature Biotechnology demonstrating potent in vivo gene editing of hematopoietic stem cells (HSCs) in the bone marrow with systemic delivery in preclinical murine models using fusogen technology:

The article describes a study that evaluated a systemically delivered virus-like particle (VLP) using the fusogen technology to target and gene edit HSCs in vivo. Results show potent and cell-specific in vivo delivery and gene editing of HSCs in the bone marrow in several murine models, with stable gene editing of long-term HSCs.
This broadens the application of fusogen technology beyond T cells to a second cell type, HSCs, and underscores the ability to deliver diverse payloads, including CRISPR gene-editing and base-editing machinery.

Raised aggregate gross proceeds of $133.7 million from sales of common stock through Sana’s at-the-market offering facility (ATM) and equity financing in 2025; expected cash runway into late 2026.

Closed public offering in August 2025 of 24.3 million shares of Sana’s common stock, including 3.4 million shares pursuant to the full exercise of the underwriters’ option to purchase additional shares, and pre-funded warrants to purchase 1.5 million shares of Sana’s common stock. The gross proceeds from this offering were $86.3 million before deducting underwriting discounts and commissions and offering expenses.
Raised gross proceeds of $47.4 million in 2025 from sales of common stock through Sana’s ATM.

Strengthened leadership with the appointment of new Chief Financial Officer

In the first quarter of 2026, appointed Brian Piper as Executive Vice President, Chief Financial Officer. Mr. Piper has decades of experience in financial management within the biotechnology sector – including CFO roles at Scorpion Therapeutics, Antares Therapeutics, and Prelude Therapeutics – and has successfully led financings and worked with companies to maximize their assets.

Fourth Quarter 2025 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of December 31, 2025 were $138.4 million compared to $152.5 million as of December 31, 2024. The decrease of $14.1 million was primarily driven by cash used in operations of $143.8 million, partially offset by net proceeds from equity financings of $126.4 million, proceeds from stock option exercises and Sana’s employee stock purchase plan of $2.7 million, and other cash inflows.
Research and Development Expenses: For the three and twelve months ended December 31, 2025, research and development expenses, inclusive of non-cash expenses, were $34.9 million and $132.0 million, respectively, compared to $45.1 million and $215.7 million for the same periods in 2024. The decreases of $10.2 million and $83.7 million for the three and twelve months ended December 31, 2025 compared to the same periods in 2024, respectively, were primarily due to the portfolio prioritization announced in the fourth quarter of 2024, which resulted in a reduction of the scope of research, laboratory, and clinical development activities, lower personnel-related costs, including non-cash stock-based compensation, and a decrease in facility and other allocated costs. Research and development expenses include non-cash stock-based compensation of $3.2 million and $15.2 million, respectively, for the three and twelve months ended December 31, 2025, and $3.9 million and $23.4 million for the same periods in 2024.
Research and Development Related Success Payments and Contingent Consideration: For the three and twelve months ended December 31, 2025, Sana recognized non-cash expenses of $14.1 million and $29.4 million, respectively, compared to non-cash gains of $13.4 million and $8.9 million for the same periods in 2024, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate. The value of these potential liabilities fluctuates significantly with changes in Sana’s market capitalization and stock price.
General and Administrative Expenses: General and administrative expenses for the three and twelve months ended December 31, 2025, inclusive of non-cash expenses, were $12.2 million and $44.3 million, respectively, compared to $17.3 million and $64.0 million for the same periods in 2024. The decrease of $5.1 million for the three months ended December 31, 2025 compared to the same period in 2024 was primarily due to personnel-related costs incurred in connection with the portfolio prioritization announced in the fourth quarter of 2024. The decrease of $19.7 million for the twelve months ended December 31, 2025 compared to the same period in 2024 was primarily due to lower personnel-related costs, including non-cash stock-based compensation, due to a decrease in headcount in connection with the portfolio prioritization announced in the fourth quarter of 2024, and decreased legal and consulting fees. General and administrative expenses include non-cash stock-based compensation of $3.2 million and $10.3 million for the three and twelve months ended December 31, 2025, respectively, compared to $2.5 million and $14.3 million for the same periods in 2024.
Impairment of Long-Lived Assets: For the twelve months ended December 31, 2025, non-cash impairment of long-lived assets was $44.6 million, compared to $1.9 million for the same period in 2024. The non-cash impairment of $44.6 million recorded in the second quarter of 2025 was primarily related to Sana’s manufacturing facility in Bothell, Washington and certain laboratory and office space in Seattle, Washington. Because of the increased availability of manufacturing capacity at third-party contract development and manufacturing organizations (CDMOs) for cell and gene therapy products as well as progress in understanding its near-term manufacturing needs, Sana expects to use CDMOs to meet its manufacturing needs at present and has suspended further build-out of internal manufacturing capabilities. Impairment of long-lived assets in 2024 consists of non-cash losses recognized for the impairment of certain laboratory equipment and leasehold improvements as a result of the portfolio prioritization in the fourth quarter of 2024.
Net Loss: Net loss for the three and twelve months ended December 31, 2025 was $58.8 million, or $0.21 per share, and $244.2 million, or $0.96 per share, respectively, compared to $49.1 million, or $0.21 per share, and $266.8 million, or $1.16 per share, for the same periods in 2024.

(Press release, Sana Biotechnology, MAR 3, 2026, View Source [SID1234663231])