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])

On March 3, 2026 Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, reported financial results for the fourth quarter and full year ended December 31, 2025, and provided an update on recent company developments.

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"Our highest priority is to serve children and adults living with SMA by bringing apitegromab through the regulatory review process as quickly as possible," said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. "To that end, we are encouraged by the FDA’s continued engagement and shared sense of urgency as Novo Nordisk works expeditiously to remediate its Catalent Indiana facility. We are ready to resubmit our apitegromab BLA following successful reinspection of the site by the FDA."

Mr. Hallal continued, "As we prepare to usher in the next phase of innovation for patients with SMA, we continue to strengthen our financial position while aggressively advancing our pipeline and expect 2026 to be a transformative year for Scholar Rock."

Business Highlights and Upcoming Milestones

Apitegromab

Apitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. It is the first and only muscle-targeted therapeutic candidate in spinal muscular atrophy (SMA) to demonstrate a statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial (SAPPHIRE).

SMA Program

● BLA resubmission and U.S. launch, following approval, expected in 2026. A meeting between FDA and Catalent Indiana occurred early in the first quarter of 2026. The meeting was constructive and included a discussion of Novo Nordisk’s progress remediating the Catalent Indiana facility. No additional corrective actions were requested by FDA. Scholar Rock plans to resubmit the apitegromab BLA following a successful reinspection of the site.
● U.S. commercial team preparing for launch. The commercial team is expanding its reach and deepening relationships with key stakeholders, including SMA treatment centers and payers. The team’s focus includes educating on the importance of addressing the full motor unit, which consists of the motor neuron and the muscle.
● European Medicines Agency (EMA) regulatory review ongoing. A decision by EMA on the apitegromab Marketing Authorisation Application (MAA) is expected in mid-2026. The European team continues to engage with key stakeholders on SMA disease awareness and education initiatives. The Company is planning for an apitegromab launch in Europe in the second half of 2026, beginning with Germany.
● Advancing key activities at second fill-finish facility. Technology transfer continues at a second U.S.-based fill-finish facility to strengthen supply continuity and support future commercial demand. Engineering runs are underway with additional manufacturing runs planned through the second quarter of 2026. Scholar Rock expects to submit a supplemental BLA (sBLA) for this fill-finish facility later in 2026.
● Phase 2 OPAL clinical trial ongoing. Enrollment and patient dosing continue in the Phase 2 OPAL study (NCT07047144). The trial is designed to evaluate apitegromab in infants and toddlers with SMA under two years of age who have received an approved SMN1-targeted gene therapy or who are receiving ongoing treatment with an approved SMN2-targeted therapy.
● Development activities for subcutaneous apitegromab progressing. Scholar Rock is advancing a subcutaneous formulation of apitegromab intended to provide optionality for patients as a small volume, self- or caregiver-administered anti-myostatin antibody suitable for an autoinjector. A Phase 1 study in healthy volunteers has been completed, and further development activities are ongoing, including planned FDA and EMA regulatory engagements.
FSHD Program

● Phase 2 FORGE trial on track for initiation in mid-2026. Scholar Rock is developing apitegromab for the treatment of people with facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare, progressive neuromuscular disease characterized by muscle atrophy and functional decline, affecting approximately 30,000 individuals across the U.S. and Europe. The IND application is cleared, and the Company continues to anticipate the initiation of a Phase 2 randomized, double-blind, placebo-controlled trial, called FORGE, in mid-2026.
SRK-439

SRK-439 is a novel, investigational, subcutaneously administered myostatin inhibitor that binds to pro- and latent myostatin with high affinity and selectivity (i.e., no GDF11 or Activin A binding). Based on preclinical data, SRK-439 has the potential to potently inhibit myostatin and increase muscle mass.

● Dosing continues in Phase 1 healthy volunteer study. A Phase 1 study evaluating SRK-439 in healthy volunteers is underway, with topline data expected in the second half of 2026.
Corporate Update

● Secured new debt facility for up to $550 million in non-dilutive capital from funds managed by Blue Owl Capital (NYSE: OWL). This debt facility is expected to support commercialization of apitegromab and strategic advancement of key pipeline programs. The debt facility matures in February 2032, and consists of the following:
o

$100 million, which became available at closing and was used to retire Scholar Rock’s prior debt facility with Oxford Finance;

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An additional $100 million to be drawn down in the first quarter of 2026;

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Up to $150 million available upon FDA approval of apitegromab; and

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An option for additional incremental facilities of up to $200 million at the mutual consent of Scholar Rock and Blue Owl Capital.

Fourth Quarter and Full Year 2025 Financial Results

Scholar Rock reported a net loss of $91.0 million, including stock-based compensation of $19.4 million, for the quarter ended December 31, 2025, compared to a net loss of $66.5 million, including stock-based compensation of $9.5 million, for the quarter ended December 31, 2024. Net loss per common share was $0.88 for the quarter ended December 31, 2025, compared to $0.61 per common share for the quarter ended December 31, 2024. For the full year ended December 31, 2025, Scholar Rock reported a net loss of $377.9 million, including stock-based compensation of $75.6 million, compared to a net loss of $246.3 million for the year ended December 31, 2024, including stock-based compensation of $36.6 million. Net loss per common share was $3.29 for the full year ended December 31, 2025, compared to $2.47 per common share for the full year ended December 31, 2024.

● The Company did not record any revenue for the quarters ended December 31, 2025 and 2024, or for the full years ended December 31, 2025 and 2024.
● Research and development expense was $46.9 million, including $5.3 million in stock-based compensation, for the quarter ended December 31, 2025, compared to $50.4 million, including $4.0 million in stock-based compensation, for the quarter ended December 31, 2024. For the full year ended December 31, 2025, research and development expense was $208.4 million, including $20.7 million in stock-based compensation, compared to $184.5 million, including $16.0 million in stock-based compensation, for the full year ended December 31, 2024.
● General and administrative expense was $45.0 million, including $14.1 million in stock-based compensation, for the quarter ended December 31, 2025, compared to $19.0 million, including $5.5 million in stock-based compensation, for the quarter ended December 31, 2024. For the full year ended December 31, 2025, general and administrative expense was $176.2 million, including $54.9 million in stock-based compensation, compared to $67.5 million, including $20.6 million in stock-based compensation, for the full year ended December 31, 2024.
● As of December 31, 2025, Scholar Rock had cash, cash equivalents, and marketable securities of $367.6 million. This reflects $60.4 million from the exercise of outstanding warrants for the quarter ended December 31, 2025.

Conference Call Information

Scholar Rock will host a conference call and webcast today, Tuesday, March 3, at 8:00 a.m. ET to review its fourth quarter and full year 2025 financial results and discuss recent business updates. To access the live audio webcast, please go to "Events and Presentations" in the Investors section of the Scholar Rock website at View Source

To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call. A replay of the webcast will be available on the Company’s website for approximately 90 days.

(Press release, Scholar Rock, MAR 3, 2026, View Source [SID1234663232])

On March 3, 2026 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel product candidates for immuno-inflammatory diseases, reported that on Tuesday March 10, 2026, at 1:40 PM EDT, Aclaris’ Chief Executive Officer Dr. Neal Walker and other members of Aclaris’ senior leadership team will participate in a fireside chat during the Leerink Partners 2026 Global Healthcare Conference in Miami, FL.

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A live and archived webcast of this event will be accessible on the Events page of the Aclaris website, www.aclaristx.com. The webcasts will be available on the Aclaris website for at least 30 days.

(Press release, Aclaris Therapeutics, MAR 3, 2026, View Source [SID1234663217])

On March 2, 2026 Defence Therapeutics Inc. ("Defence" or the "Company"), a publicly traded biotechnology company advancing next-generation therapeutics using its proprietary Accum platform, reported its participation in a series of major international industry events taking place in March 2026.

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These high-profile meetings will provide Defence with multiple opportunities to advance strategic partnerships, strengthen its global network, and further position Accum as a differentiated intracellular delivery technology for antibody-drug conjugates (ADCs), radiopharmaceutical conjugates (RDCs), and other complex biologics.

BIO-Europe Spring: Primary Focus on Strategic Partnerships

Defence will participate in BIO-Europe Spring (March 23–26, Lisbon, Portugal), the premier global partnering event for the life sciences industry. At BIO-Europe, the Company will prioritize discussions with biopharmaceutical partners seeking to collaborate on enhancing the intracellular delivery and efficacy of their ADC programs using Accum.

DCAT Week: Advancing Biomanufacturing Capabilities

Defence will also attend DCAT Week (March 23–26, New York), where the Company will focus on strengthening its manufacturing strategy. During DCAT, Defence plans to meet with contract research and manufacturing partners to advance the robust and high-quality biomanufacturing of Accum, a key operational objective for the Company. Defence has recently become a member of the Drug, Chemical & Associated Technologies Association ("DCAT"), reinforcing its commitment to ensuring the well-characterized, quality-controlled, and commercially ready production of Accum.

Expanding U.S. Presence and Investor Engagement

In parallel, Defence will participate in the South Florida Life Sciences Showcase (March 24, Miami) and FII PRIORITY Miami (March 25–27), supporting the Company’s ongoing investor outreach and business development efforts in the United States. Defence has also recently joined BioFlorida, further strengthening its footprint within the Florida life sciences ecosystem.

"As we engage with partners and industry stakeholders across these key events, our priority is to build meaningful collaborations that support the continued growth and adoption of Accum," said Sébastien Plouffe, CEO of Defence Therapeutics. "By enabling more efficient intracellular delivery of complex biologics, Accum has the potential to significantly enhance therapeutic performance and tolerability, positioning the platform as a game-changing solution in the ADC field.". To explore partnering opportunities or schedule a meeting, please contact Defence Therapeutics at [email protected].

(Press release, Defence Therapeutics, MAR 2, 2026, View Source;utm_medium=rss&utm_campaign=defence-therapeutics-to-showcase-accum-platform-at-key-international-industry-events-in-march [SID1234663176])

On March 2, 2026 Nuvation Bio Inc. (NYSE: NUVB), a global oncology company focused on tackling some of the toughest challenges in cancer treatment, reported financial results for the fourth quarter and full year ended December 31, 2025, and provided a business update.

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"We ended our transformational 2025 with a strong fourth quarter that underscored IBTROZI’s rapid adoption, and we believe our medicine is becoming the new standard of care for people living with advanced ROS1+ NSCLC across treatment lines. In 2026, we look forward to further building upon our successful U.S. launch, and partnering closely with Eisai to bring IBTROZI to more patients around the globe," said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. "We are also excited and eager to progress our promising pipeline, with safusidenib now being studied in the pivotal Phase 3 SIGMA trial across those living with high-risk and high-grade IDH1-mutant glioma where significant needs exist for these patients. Lastly, we continue to evaluate additional internal preclinical candidates and external business development opportunities in hopes of furthering our mission to tackle some of the toughest challenges in cancer treatment."

Fourth Quarter 2025 and Recent Corporate Highlights:

IBTROZI (taletrectinib), ROS1 inhibitor: Advanced ROS1+ NSCLC

In the fourth quarter of 2025, 216 new patients started treatment on IBTROZI for advanced ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC), reflecting the high rate of adoption and confidence in IBTROZI among healthcare professionals and patients.
With 432 new patients started since launch in the second half of June 2025, the treatment adoption rate is approximately six times greater than that of prior recent ROS1 tyrosine kinase inhibitor (TKI) launches based on IQVIA data.
On January 11, 2026, the Company entered an exclusive license and collaboration agreement with Eisai Co., Ltd. to develop, register and commercialize taletrectinib for the treatment of ROS1+ NSCLC in Europe, the Middle East, North Africa, Russia, Turkey, Canada, Australia, New Zealand, Singapore, the Philippines, Indonesia, Thailand, Malaysia, Vietnam, and India.
In the fourth quarter of 2025, the Company received a $25 million milestone payment from Nippon Kayaku as a result of establishing the reimbursement price in Japan.
In October 2025, the Company presented clinical data from the pivotal TRUST-II study evaluating IBTROZI in patients previously treated with entrectinib, a brain-penetrant ROS1 therapy at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025.
IBTROZI demonstrated a confirmed overall response rate of 80% in 10 patients whose tumors progressed following treatment with entrectinib.
Safusidenib, mIDH1 inhibitor: IDH1-mutant glioma

In December 2025, the Company announced the publication of positive results from a Phase 2 study of safusidenib in patients with chemotherapy- and radiotherapy-naïve grade 2 IDH1-mutant gliomas. The findings were first published online on November 8, 2025, in Neuro-Oncology.
Safusidenib demonstrated durable responses, with an ORR of 44% and 88% of patients were progression-free at 24 months.
The findings supported favorable interactions with the U.S. Food and Drug Administration (FDA) and the now finalized protocol amendment making the SIGMA study (G203) a Phase 3, pivotal trial evaluating safusidenib maintenance treatment in high-risk and high-grade IDH1-mutant gliomas. The study now also includes an additional exploratory cohort in grade 3 oligodendroglioma.
In October 2025, the Company enrolled the first patient in the global SIGMA study (G203).
Fourth Quarter and Full Year 2025 Financial Results
As of December 31, 2025, Nuvation Bio had cash, cash equivalents, and marketable securities of $529.2 million.

Product Revenue, Net
To date, our only source of product revenue has been from the U.S. sales of IBTROZI. We began distributing IBTROZI to our U.S. customers in June 2025. Net product revenue from U.S. sales of IBTROZI was approximately $15.7 million and $24.7 million for the three and twelve months ended December 31, 2025, respectively.

Collaboration and License Agreements Revenue
For the three months ended December 31, 2025, collaboration and license agreements revenue was $26.2 million, compared to $5.7 million for the three months ended December 31, 2024. The increase is primarily due to a $25 million milestone payment from Nippon Kayaku as a result of establishment of the reimbursement price for IBTROZI in Japan in Q4, offset by a $4.5 million decrease in research and development service revenue under the collaboration agreement with Innovent. Taletrectinib was included in China’s National Reimbursement Drug List effective January 1, 2026.

For the twelve months ended December 31, 2025, collaboration and license agreements revenue was $38.2 million, compared to $7.9 million for 2024. The increase is primarily due to a $19.1 million increase in license revenue and a $6.3 million increase in research and development service revenue as a result of milestone payment from Nippon Kayaku for the establishment of the reimbursement price in Japan in December 2025, a $3.6 million increase in product supply revenue, and a $1.3 million increase in royalty revenue.

Research and Development Expenses
For the three months ended December 31, 2025, research and development expenses were $34.3 million, compared to $29.3 million for the three months ended December 31, 2024. The increase was due to an $11.4 million increase in third-party costs related to clinical studies, offset by a $2.4 million decrease in personnel-related costs because employee compensation and benefit costs directly related to commercial drug production were capitalized into inventory, as well as a $4.0 million decrease in regulatory milestone payments to Daiichi.

For the twelve months ended December 31, 2025, research and development expenses were $115.1 million, compared to $99.1 million for the twelve months ended December 31, 2024. The increase was primarily due to a $7.8 million increase in salaries and other benefits driven by the increase in headcount and stock-based compensation primarily related to one-time charge for the vesting of performance-based awards upon receiving U.S. FDA approval of IBTROZI, a $12.1 million increase in third-party costs related to clinical studies, and a $0.1 million increase in amortization of assembled workforce, offset by a $4.0 million decrease in regulatory milestone payments to Daiichi.

Acquired In-process Research and Development Expenses
On April 9, 2024, as a result of the acquisition of AnHeart Therapeutics Ltd., we recorded a $425.1 million charge representing an acquired in-process research and development asset with no alternative future use in acquired in-process research and development expenses.

Selling, General and Administrative Expenses
For the three months ended December 31, 2025, selling, general, and administrative expenses were $40.3 million, compared to $26.1 million for the three months ended December 31, 2024. The increase was due to a $7.3 million increase in personnel-related costs as a result of the increase in headcount, a $5.9 million increase in sales and marketing expenses, a $2.1 million increase in legal fees, offset by a $0.4 million decrease in professional fees, and a $0.7 million decrease in foreign currency impact.

For the twelve months ended December 31, 2025, selling, general, and administrative expenses were $151.6 million, compared to $69.2 million for the twelve months ended December 31, 2024. The increase was due to a $39.4 million increase in personnel-related costs as a result of the increase in headcount and stock-based compensation primarily related to one-time charge for the vesting of performance-based awards upon receiving U.S. FDA approval of IBTROZI, a $41.0 million increase in sales and marketing expenses, a $3.1 million increase in legal fees, a $0.1 million increase in professional fees and a $0.3 million increase in other expenses, offset by a $1.5 million decrease in foreign currency impact.

Net loss
For the three months ended December 31, 2025, Nuvation Bio reported a net loss of $36.6 million, or $(0.11) per share. The net loss for the comparable period in 2024 was $49.4 million, or $(0.15) per share.

For the twelve months ended December 31, 2025, Nuvation Bio reported a net loss of $204.6 million, or $(0.60) per share. The net loss for the comparable period in 2024 was $567.9 million, or $(2.11) per share.

Conference Call and Webcast
Nuvation Bio will host a conference call and webcast today, March 2, 2026, at 4:30 pm ET to discuss its financial results for the fourth quarter and full year of 2025 and provide business updates.

Investors and the general public are invited to listen to the live webcast and may register on the Investor Relations section of the Nuvation Bio website. To access the live conference call, participants can dial +1 833-470-1428 (U.S. toll-free) and enter access code 833155. An archived recording will be available on Nuvation Bio’s website for 90 days following the event.

About ROS1+ NSCLC
Each year, more than one million people globally are diagnosed with non-small cell lung cancer (NSCLC), the most common form of lung cancer. It is estimated that approximately 2% of patients with NSCLC have ROS1+ disease. About 35% of patients newly diagnosed with metastatic ROS1+ NSCLC have tumors that have spread to their brain. The brain is also the most common site of disease progression, with about 50% of previously treated patients developing central nervous system (CNS) metastases.

About IBTROZI
IBTROZI is an oral, potent, CNS-active, selective, next-generation ROS1 inhibitor therapy. On June 11, 2025, following Priority Review and Breakthrough Therapy designations for both TKI-naive and TKI-pretreated disease, the U.S. Food and Drug Administration (FDA) approved IBTROZI for the treatment of adult patients with locally advanced or metastatic ROS1+ NSCLC. Learn more at IBTROZI.com.

About the TRUST Clinical Program
The TRUST clinical program comprises three registrational studies evaluating the safety and efficacy of IBTROZI. TRUST-I (NCT04395677) and TRUST-II (NCT04919811) are Phase 2 single-arm studies evaluating IBTROZI for the treatment of adults with advanced ROS1+ NSCLC in China (N=173) and globally (N=189), respectively. The primary endpoint of both studies is confirmed objective response rate (cORR) as assessed by an independent review committee. TRUST-IV (NCT07154706) is a Phase 3 placebo-controlled study evaluating IBTROZI for the adjuvant treatment of adults with resected early-stage ROS1+ NSCLC. The study will enroll approximately 180 patients in the U.S., Canada, Europe, Japan and China. The primary endpoint is disease-free survival as determined by investigator, and the primary completion date is estimated to be in 2030. Nuvation Bio is also sponsoring TRUST-III (NCT06564324), a confirmatory randomized Phase 3 study evaluating IBTROZI versus crizotinib in 138 patients in China with advanced ROS1+ NSCLC who have not previously received ROS1 TKIs.

Indication
IBTROZI is indicated for the treatment of adult patients with locally advanced or metastatic ROS1+ non-small cell lung cancer (NSCLC).

IMPORTANT SAFETY INFORMATION FOR IBTROZI (taletrectinib)

WARNINGS AND PRECAUTIONS

Hepatotoxicity: Hepatotoxicity, including drug-induced liver injury and fatal adverse reactions, can occur. 88% of patients experienced increased AST, including 10% Grade 3/4. 85% of patients experienced increased ALT, including 13% Grade 3/4. Fatal liver events occurred in 0.6% of patients. Median time to first onset of AST or ALT elevation was 15 days (range: 3 days to 20.8 months).

Increased AST or ALT each led to dose interruption in 7% of patients and dose reduction in 5% and 9% of patients, respectively. Permanent discontinuation was caused by increased AST, ALT, or bilirubin each in 0.3% and by hepatotoxicity in 0.6% of patients.

Concurrent elevations in AST or ALT ≥3 times the ULN and total bilirubin ≥2 times the ULN, with normal alkaline phosphatase, occurred in 0.6% of patients.

Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, or fatal ILD or pneumonitis can occur. ILD/pneumonitis occurred in 2.3% of patients, including 1.1% Grade 3/4. One fatal ILD case occurred at the 400 mg daily dose. Median time to first onset of ILD/pneumonitis was 3.8 months (range: 12 days to 11.8 months).

ILD/pneumonitis led to dose interruption in 1.1% of patients, dose reduction in 0.6% of patients, and permanent discontinuation in 0.6% of patients.

QTc Interval Prolongation: QTc interval prolongation can occur, which can increase the risk for ventricular tachyarrhythmias (e.g., torsades de pointes) or sudden death. IBTROZI prolongs the QTc interval in a concentration-dependent manner.

In patients who received IBTROZI and underwent at least one post baseline ECG, QTcF increase of >60 msec compared to baseline and QTcF >500 msec occurred in 13% and 2.6% of patients, respectively. 3.4% of patients experienced Grade ≥3. Median time from first dose of IBTROZI to onset of ECG QT prolongation was 22 days (range: 1 day to 38.7 months). Dose interruption and dose reduction each occurred in 2.8% of patients.

Significant QTc interval prolongation may occur when IBTROZI is taken with food, strong and moderate CYP3A inhibitors, and/or drugs with a known potential to prolong QTc. Administer IBTROZI on an empty stomach. Avoid concomitant use with strong and moderate CYP3A inhibitors and/or drugs with a known potential to prolong QTc.

Hyperuricemia: Hyperuricemia can occur and was reported in 14% of patients, with 16% of these requiring urate-lowering medication without pre-existing gout or hyperuricemia. 0.3% of patients experienced Grade ≥3. Median time to first onset was 2.1 months (range: 7 days to 35.8 months). Dose interruption occurred in 0.3% of patients.

Myalgia with Creatine Phosphokinase (CPK) Elevation: Myalgia with or without CPK elevation can occur. Myalgia occurred in 10% of patients. Median time to first onset was 11 days (range: 2 days to 10 months).

Concurrent myalgia with increased CPK within a 7-day time period occurred in 0.9% of patients. Dose interruption occurred in 0.3% of patients with myalgia and concurrent CPK elevation.

Skeletal Fractures: IBTROZI can increase the risk of fractures. ROS1 inhibitors as a class have been associated with skeletal fractures. 3.4% of patients experienced fractures, including 1.4% Grade 3. Some fractures occurred in the setting of a fall or other predisposing factors. Median time to first onset of fracture was 10.7 months (range: 26 days to 29.1 months). Dose interruption occurred in 0.3% of patients.

Embryo-Fetal Toxicity: Based on literature, animal studies, and its mechanism of action, IBTROZI can cause fetal harm when administered to a pregnant woman.

ADVERSE REACTIONS

Among patients who received IBTROZI, the most frequently reported adverse reactions (≥20%) were diarrhea (64%), nausea (47%), vomiting (43%), dizziness (22%), rash (22%), constipation (21%), and fatigue (20%).

The most frequently reported Grade 3/4 laboratory abnormalities (≥5%) were increased ALT (13%), increased AST (10%), decreased neutrophils (5%), and increased creatine phosphokinase (5%).

DRUG INTERACTIONS

Strong and Moderate CYP3A Inhibitors/CYP3A Inducers and Drugs that Prolong the QTc Interval: Avoid concomitant use.
Gastric Acid Reducing Agents: Avoid concomitant use with PPIs and H2 receptor antagonists. If an acid-reducing agent cannot be avoided, administer locally acting antacids at least 2 hours before or 2 hours after taking IBTROZI.
OTHER CONSIDERATIONS

Pregnancy: Please see important information in Warnings and Precautions under Embryo-Fetal Toxicity.
Lactation: Advise women not to breastfeed during treatment and for 3 weeks after the last dose.
Effect on Fertility: Based on findings in animals, IBTROZI may impair fertility in males and females. The effects on animal fertility were reversible.
Pediatric Use: The safety and effectiveness of IBTROZI in pediatric patients has not been established.
Photosensitivity: IBTROZI can cause photosensitivity. Advise patients to minimize sun exposure and to use sun protection, including broad-spectrum sunscreen, during treatment and for at least 5 days after discontinuation.
Please see accompanying full Prescribing Information.

About IDH1-Mutant Glioma
Gliomas are the most common type of brain cancer in adults worldwide. In the U.S., nearly 2,400 people are diagnosed with IDH1-mutant gliomas each year. Most patients are diagnosed in their 30s and 40s. While patients with IDH1 mutations generally have longer survival times than those with wild-type IDH1, gliomas are not currently curable and prognosis worsens for those with high grade tumors.

About Safusidenib
Safusidenib is a novel, oral, potent, brain-penetrant, targeted inhibitor of mutant IDH1. In Phase 1 and 2 clinical studies, safusidenib was well-tolerated and demonstrated anti-tumor activity and high blood-brain barrier penetration.

(Press release, Nuvation Bio, MAR 2, 2026, View Source [SID1234663192])