On August 11, 2025 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported financial results for the second quarter ended June 30, 2025, and provided a corporate update (Press release, Theriva Biologics, AUG 11, 2025, View Source [SID1234655083]).

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

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

                  Schedule Your 30 min Free Demo!

"We are highly encouraged by the progress we’ve made so far in the first half of 2025," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We have achieved a significant milestone with the positive readout from the Phase 2b VIRAGE trial, where our lead asset VCN-01 (zabilugene almadenorepvec) achieved its primary survival and safety endpoints in metastatic pancreatic cancer patients treated with first-line standard of care chemotherapy gemcitabine/nab-paclitaxel. We are now preparing a study protocol for a potential Phase 3 clinical trial and advancing VCN-01’s manufacturing scale-up using our propietary suspension cell line. Leveraging our clinical and manufacturing momentum, we have also initiated strategic outreach to identify potential partners for VCN-01’s late-stage clinical development."

Recent Highlights and Anticipated Milestones

VCN-01

Metastatic Pancreatic Ductal Adenocarcinoma (PDAC):

Positive topline results from the VIRAGE Phase 2b trial were announced in May. PDAC patients treated with VCN-01 (zabilugene almadenorepvec) plus gemcitabine/nab-paclitaxel standard-of-care (SoC) chemotherapy had increased overall survival (OS), progression free survival (PFS), and duration of response (DOR) compared to patients treated with gemcitabine/nab-paclitaxel SoC.
Theriva hosted a virtual event featuring eminent pancreatic cancer clinician/researchers to review and discuss the data from the VIRAGE trial of VCN-01. To access the replay of the event, click HERE.
Theriva and VIRAGE trial investigators participated in an off-site meeting during the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 Annual Meeting to review the topline data and discuss the conclusions relevant to the design and execution of a potential Phase 3 clinical trial in metastatic pancreatic ductal adenocarcinoma (PDAC).
Expanded data from the VIRAGE trial are to be presented at a mini oral session at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2025 Congress. The presentation entitled "VIRAGE trial: randomized Phase IIb, open-label, study of Nab-Paclitaxel and Gemcitabine with/without intravenous VCN-01 in Patients with Metastatic Pancreatic Cancer (mPDAC)" (abstract 2216MO) is planned to be presented on Monday, October 20, 2025 by Dr. Rocío García-Carbonero, Hospital 12 de Octubre, Madrid, Spain and subsequently reviewed by an invited Discussant.
Retinoblastoma:

As previously announced, safety and clinical outcomes of Phase 1 study (NCT03284268) of VCN-01 (zabilugene almadenorepvec) in refractory retinoblastoma patients were presented during a poster session at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, Illinois on Saturday, May 31, 2025. The abstract can be viewed here.
Second Quarter Ended June 30, 2025 Financial Results

General and administrative expenses increased to $11.2 million for the three months ended June 30, 2025, from $1.5 million for the three months ended June 30, 2024. This increase of 662% is primarily comprised of the increase in fair value of the contingent consideration adjustment of $9.2 million due to the VIRAGE Phase 2b clinical trial of VCN-01 in PDAC achieving its primary endpoint and increased registration fees. The charge related to stock-based compensation expense was $97,000 for the three months ended June 30, 2025, compared to $114,000 for the three months ended June 30, 2024.

Research and development expenses decreased to $2.0 million for the three months ended June 30, 2025, from approximately $3.0 million for the three months ended June 30, 2024. This decrease of 34% is primarily the result of lower clinical trial expenses related to the Company’s VIRAGE Phase 2b clinical trial of VCN-01 in PDAC, lower indirect cost related to decreased VCN-01 manufacturing costs and lower clinical trial expenses related to its Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, offset by higher patent expenses related to SYN-020. Theriva anticipates research and development expense to increase as it completes its VIRAGE Phase 2b clinical trial of VCN-01 and plans for its potential Phase 3 clinical trial of VCN-01 in PDAC, advances its VCN-01 program in retinoblastoma, expands GMP scale-up manufacturing activities for VCN-01, and continues supporting the Company’s other preclinical and discovery initiatives. The charge related to stock-based compensation expense was $76,000 for the three months ended June 30, 2025, compared to $58,000 related to stock-based compensation expense for the three months ended June 30, 2024.

Other income was $74,000 for the three months ended June 30, 2025 compared to other income of $172,000 for the three months ended June 30, 2024. Other income for the three months ended June 30, 2025 is primarily comprised of interest income of $54,000 and an exchange gain of $20,000. Other income for the three months ended June 30, 2024 is primarily comprised of interest income of $173,000 and exchange loss of $1,000.

Cash and cash equivalents totaled $12.1 million as of June 30, 2025, compared to $11.6 million as of December 31, 2024. The Company expects that its cash position of $9.5 million as of early August 2025, will be able to fund its operations into the first quarter of 2026.

About VCN-01

VCN-01 (zabilugene almadenorepvec) is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to 142 patients to date in clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.

About Pancreatic Ductal Adenocarcinoma

Cancer of the pancreas consists of two main histological types: cancer that arises from the ductal (exocrine) cells of the pancreas or, much less often, cancers may arise from the endocrine compartment of the pancreas. Pancreatic ductal adenocarcinoma ("PDAC") accounts for more than 90% of all pancreatic tumors. It can be located either in the head of the pancreas or in the body/tail. Pancreatic cancer usually metastasizes to the liver and peritoneum. Other less common metastatic sites are the lungs, brain, kidney and bone. In its early stages, pancreatic cancer does not typically result in any characteristic symptoms, so in most cases it is diagnosed in its late stages (locally advanced non-metastatic or metastatic disease) when surgical resection and possibly curative treatment is not possible. It is generally assumed that only 10% of cases are resectable at presentation, whereas 30-40% of patients are diagnosed at local advanced/unresectable stage and 50-60% present with distant metastases.

About VIRAGE

VIRAGE was a two-arm, Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. Patients were enrolled at 5 sites in the U.S. and 9 sites in Spain. In both the control and VCN-01 (zabilugene almadenorepvec) treatment arms, patients received gemcitabine/nab-paclitaxel standard-of-care chemotherapy in repeated 28-day cycles until disease progression. In the VCN-01 treatment arm only, patients were also administered intravenous VCN-01 seven-days prior to starting the first and fourth cycles of gemcitabine/nab-paclitaxel treatment (study days 1 and ~92 respectively). Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, duration of response, and measures of VCN-01 biodistribution, replication, and immune response. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).

About Retinoblastoma

Retinoblastoma is a tumor that originates in the retina and is the most common type of eye cancer in children. It occurs in approximately 1/14,000 – 1/18,000 live newborns and accounts for 15% of the tumors in the pediatric population < 1 year old. The average age of pediatric patients at diagnosis is 2, and it rarely occurs in children older than 6. In Europe, retinoblastoma has an estimated incidence rate of 1 per 13,844 live births (14.1 per million children under the age of 5) with approximately 300 children diagnosed per year (Stacey et al. 2021). Preserving life and preventing the loss of an eye, blindness, and other serious effects of treatment that reduce the patient’s life span or the quality of life remains a challenge. In addition, children with retinoblastoma have been more likely to lose their eye and die of metastatic disease in low-resource countries.

On August 11, 2025 Aptevo Therapeutics Inc. (Nasdaq:APVO), a clinical-stage biotechnology Company focused on developing novel immune-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported financial results for the quarter ended June 30, 2025, and provided a business update (Press release, Aptevo Therapeutics, AUG 11, 2025, View Source [SID1234655082]).

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

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

                  Schedule Your 30 min Free Demo!

Second Quarter Highlights

Pipeline Expansion – Introduced APVO455, a Nectin-4 x CD3 bispecific for solid tumors, expanding the Company’s suite of differentiated CRIS-7-derived CD3 – engaging molecules in development, including lead candidate, mipletamig

Mipletamig for acute myeloid leukemia (AML) – Achieved 85% remission* rate (11/13 patients) in frontline AML patients across two trials in combination therapy

Increased Liquidity – Completed financings that raised a total of $15.9 million, extending cash runway into late 4Q25

Secured an equity line of credit for up to an additional $25 million in capital

*Remission = complete remission (CR) and, complete remission with blood markers that have not yet recovered (CRi).

"The second quarter marked a period of decisive progress for Aptevo across both clinical and corporate fronts," said Marvin White, President and CEO of Aptevo. We are energized by the expansion of our differentiated CD3 bispecific portfolio – anchored by mipletamig in AML and APVO442 in prostate cancer – with the introduction of APVO455, a Nectin-4 x CD3 bispecific designed to address a broad range of solid tumor types. Broadening our reach into solid tumors, beyond prostate cancer, with APVO455 underscores Aptevo’s commitment to advancing differentiated, next-generation immunotherapies in areas of significant unmet need."

Mr. White continued, "We’re pleased to report that our lead asset, mipletamig, continues to perform as designed in the clinic. In frontline acute myeloid leukemia (AML), 85% of evaluable patients achieved remission when treated in combination with standard of care across two independent trials. These encouraging results validate mipletamig’s differentiated mechanism of action and highlight its potential as a transformative therapy for AML, anchoring our CRIS-7-derived CD3 portfolio. Just as important, we strengthened our financial position with a $15.9 million raise, extending cash runway into late Q4 2025, and ensuring we are well-positioned to reach additional clinical and business milestones in the months ahead."

APVO455: A New T-Cell Engager Targeting Solid Tumors

APVO455 is a preclinical Nectin-4 x CD3 bispecific T-cell engager designed for tumors such as bladder, breast, NSCLC, and head and neck cancers, where nectin-4 is highly expressed. Unlike other approaches that restrict activity to acidic tumor environments or rely on activated T-cells, APVO455 is designed to avoid binding to or triggering T-cell activation in the periphery and do so only in the presence of nectin-4 positive tumor cells. This offers the potential for a broader therapeutic window, more consistent immune activation and the favorable safety and tolerability characteristics of Aptevo’s clinical stage CD3 engager, mipletamig.

CRIS-7-Derived CD3 Portfolio

With this APVO455 addition to the pipeline, Aptevo now has a suite of three CD3-engaging molecules in development. All three molecules share the same CRIS-7-derived CD3 binding domain in the same orientation from within our ADAPTIR/ADAPTIR-FLEX platform that we believe drives the safety results, especially regarding our intention to reduce cytokine release syndrome, as is being delivered in the clinic by mipletamig. In addition, APVO455 and APVO442 contain CD3 binding domains that are optimized for localizing to solid tumors. All three molecules are designed to drive tumor-specific immune activation while limiting harmful side effects to the patient. The suite includes:

Mipletamig, a CD123 x CD3 bispecific for frontline AML, currently in a Phase 1b/2 trial

APVO442, a PSMA x CD3 bispecific targeting prostate cancer, currently in preclinical development

And now APVO455, a Nectin-4 x CD3 bispecific developed to address multiple solid tumor types

Additional Pipeline Candidates

Aptevo continues to develop clinical candidate ALG.APV-527 ( 4-1BB x 5T4) and preclinical candidates APVO711, a checkpoint inhibitor with added functionality and APVO603, a 4-1BB x OX40 molecule. All targeting multiple solid tumor types.

Mipletamig Data Highlights

Results from the RAINIER trial, reported to date demonstrate mipletamig’s increasingly impressive clinical profile, and highlight its potential as a differentiated, highly targeted immunotherapy with a compelling safety profile. As evidence grows, the Company believes that mipletamig is emerging as a strong candidate to reshape frontline AML treatment for unfit patients and potentially advance the standard of care.

Efficacy Data Continues to Outperform Benchmarks

To date, 85% frontline patients across two trials have achieved remission

Of these remissions, multiple patients were considered to have poor prognosis at screening based on the genetic biomarkers of their disease and achieved CR, including one patient who was taken off study and successfully received a stem cell transplant. This is the most favorable outcome because transplant offers the best chance for a cure, although it is a rare occurrence in the unfit AML patient population

Stand Out Safety and Tolerability Outcomes Continue

No CRS has been reported in frontline patients treated to date

Q2 2025 Financial Highlights

Cash Position: Aptevo had cash and cash equivalents as of June 30, 2025, totaling $9.4 million. In Q2 2025, we raised $15.9M in gross proceeds from various equity offerings.

Research and Development Expenses: Research and development expenses decreased by $0.3 million, from $3.6 million for the three months ended June 30, 2024, to $3.3 million for the three months ended June 30, 2025. The decrease was primarily due to lower preclinical and ALG.APV-527 spending due to escalation phase ramping down and was offset by higher mipletamig trial costs from clinical trial patient enrollment.

General and Administrative Expenses: General and administrative expenses increased by $0.5 million, from $2.4 million for the three months ended June 30, 2024, to $2.9 million for the three months ended June 30, 2025. The increase is primarily due to higher consulting costs.

Net Loss: Aptevo had a net loss of $6.2 million or $8.40 per share for the three months ended June 30, 2025, compared to a net loss of $5.9 million or $1,236.96 per share for the corresponding period in 2024.

On August 11, 2025 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company with a pipeline of first-in-class1 targeted and immune-mediated therapeutics to fight cancer, reported financial results for the quarter ended June 30, 2025 and provided a corporate update (Press release, Tempest Therapeutics, AUG 11, 2025, View Source [SID1234655077]).

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

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

                  Schedule Your 30 min Free Demo!

"We are pleased to see the continued progress of our clinical oncology portfolio, including the recent clearance in China to initiate a pivotal trial of amezalpat combination therapy in first-line HCC, expanding on similar clearances we received in the U.S. and Europe from the FDA and EMA," said Stephen Brady, president and chief executive officer of Tempest. "We believe these milestones reflect both the promise of our therapies and the dedication of the team who brought the programs to this point. We remain actively engaged in our strategic alternatives process with the goal of maximizing value for stockholders and patients."

____________________
1 If approved by the U.S. Food and Drug Administration (FDA).

Recent Highlights

Amezalpat (TPST-1120) (clinical PPARα antagonist):
Received clearance to proceed with pivotal trial of amezalpat combination therapy for first-line HCC in China.
Granted orphan drug designation from the EMA for amezalpat for the treatment of patients with HCC.
Reported new data at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, supporting the immune component of amezalpat’s dual mechanism of action and reinforcing its potential as a novel cancer treatment.
Granted both Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) for amezalpat for the treatment of patients with HCC.
TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist):
Granted Orphan Drug designation by the FDA to treat patients with FAP.
Corporate:
Announced cost-cutting measures and plans to explore a full range of strategic alternatives to advance the company’s promising clinical-stage programs and maximize stockholder value.
Strengthened cash position with completion of $4.6 million registered direct offering of common stock in June 2025.
Financial Results

Second Quarter 2025

Tempest ended the quarter with $14.3 million in cash and cash equivalents, compared to $30.3 million on December 31, 2024. The decrease was primarily due to cash used in operating activities, offset by $4.1 million in net proceeds from the June 2025 registered direct offering, as well as $2.8 million in net proceeds from the company’s at-the-market offering program.
Net loss and net loss per share for the quarter were $7.9 million and $2.07, respectively, compared to $9.6 million and $5.52, respectively, for the same period in 2024.
Research and development expenses for the quarter were $3.9 million, compared to $5.8 million for the same period in 2024. The $1.9 million decrease was primarily due to a decrease in costs incurred as a result of re-prioritizing efforts towards exploring strategic alternatives.
General and administrative expenses for the quarter were $4.1 million, compared to $3.7 million for the same period in 2024. The $0.4 million increase was primarily related to one-time separations costs for employees terminated during the period.
Year-to-Date

Cash used in operating activities for the six months ended June 30, 2025 was $16.5 million.
Net loss and net loss per share for the six months ended June 30, 2025 were $18.7 million and $5.17, respectively, compared to $17.5 million and $10.15, respectively, for the same period in 2024.
Research and development expenses for the six months ended June 30, 2025 were $11.5 million, compared to $10.2 million for the same period in 2024. The $1.3 million increase was primarily due to an increase in costs incurred from contract research and manufacturing organizations in preparation for the company’s pivotal Phase 3 trial of amezalpat for the treatment of first-line HCC.
General and administrative expenses for the six months ended June 30, 2025 were $7.4 million, compared to $7.4 million for the same period in 2024, and were primarily related to employee compensation costs, inclusive of one-time separation costs for employees terminated during the quarter ended June 30, 2025, as well as consulting, professional services and facilities costs.

On August 11, 2025 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the second quarter 2025 (Press release, Sana Biotechnology, AUG 11, 2025, View Source [SID1234655076]).

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

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

                  Schedule Your 30 min Free Demo!

"We are pleased with the progress with the type 1 diabetes program, including recent FDA feedback on our HIP-edited GMP master cell bank and non-clinical testing plan for SC451, positive UP421 6-month clinical results presented at an invited talk at the American Diabetes Association Annual Meeting and World Transplant Congress 2025, and UP421 3-month clinical results published in the New England Journal of Medicine," said Steve Harr, Sana’s President and Chief Executive Officer. "Type 1 diabetes affects over 9 million people worldwide, and we are positioned to deliver on our goal of a broadly accessible single treatment with no immunosuppression leading to long-term normal blood glucose without exogenous insulin in patients with type 1 diabetes. We expect to file the IND for SC451 as early as next year, and we also look forward to continuing progress in the rest of our pipeline. We have raised over $100 million in new capital since the end of the second quarter, strengthening our balance sheet and allowing us to continue to invest appropriately in moving our pipeline forward."

Recent Corporate Highlights

Announced 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 is 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 type 1 diabetes patients 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 6 months 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 a mixed meal tolerance test during testing at these timepoints, consistent with insulin secretion in response to a meal. 12-week PET-MRI scanning also demonstrated islet cells at the transplant site. The study identified no safety issues, and the HIP-modified islet cells evaded immune detection.
Announced that the New England Journal of Medicine 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.
Sana and Uppsala University Hospital presented 6-month data at the 85th Annual American Diabetes Association (ADA) Scientific Sessions and the World Transplant Congress 2025, and expect to report additional data from the IST, including longer-term follow-up, as the year progresses.

Advancing our pipeline across multiple indications and modalities:

Type 1 Diabetes – The clinical study of gene-modified primary islet cells (UP421) continues to evaluate safety, survival, and function of these cells. Sana continues pre-clinical development of SC451, an O-negative, HIP-modified, iPSC-derived pancreatic islet cell therapy, and a recent FDA INTERACT meeting increases our confidence in moving forward with our HIP-edited master cell bank for GMP manufacturing and our non-clinical testing plan.
Sana expects to file an IND for SC451 as early as 2026.
Allogeneic CAR T cells – Sana is enrolling patients in both the GLEAM and VIVID trials and expects to share data in 2025.
The GLEAM trial is a Phase 1 study evaluating SC291, a HIP-modified CD19-directed allogeneic CAR T cell therapy, in patients with B-cell mediated autoimmune diseases, including refractory systemic lupus erythematosus and antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis. The VIVID trial is a Phase 1 study evaluating SC262, a HIP-modified CD22-directed allogeneic CAR T cell therapy, in patients with relapsed and/or refractory B-cell malignancies who have received prior CD19-directed CAR T therapy.
In vivo CAR T cells – SG299, which uses our fusogen platform, allows for cell-specific, in vivo delivery of various payloads. SG299 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 areas such as the liver and gonadal tissue. Sana shared data showing that an SG299 surrogate with another component can lead to deep B-cell depletion in non-human primates without the use of any lymphodepleting chemotherapy. Sana expects to file an IND for SG299 as early as 2026, and we look forward to developing it in a range of B-cell cancers and B-cell mediated autoimmune diseases.

Raised aggregate gross proceeds of approximately $105 million from sales of common stock through Sana’s at-the-market offering facility (ATM) and equity financing in July and August 2025; expected cash runway into the second half of 2026

Closed public offering in August 2025 of 20.9 million shares of Sana’s common stock and pre-funded warrants to purchase 1.5 million shares of Sana’s common stock. The gross proceeds from this offering were $75.0 million before deducting underwriting discounts and commissions and estimated offering expenses.
Raised gross proceeds of $29.5 million in July and August 2025 from sales of common stock through Sana’s ATM.

Second Quarter 2025 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of June 30, 2025 were $72.7 million compared to $152.5 million as of December 31, 2024. The decrease of $79.8 million was primarily driven by cash used in operations of $81.8 million.
Research and Development Expenses: For the three and six months ended June 30, 2025, research and development expenses, inclusive of non-cash expenses, were $29.8 million and $67.0 million, respectively, compared to $60.9 million and $117.3 million for the same periods in 2024.The decreases of $31.1 million and $50.3 million for the three and six months ended June 30, 2025 compared to the same periods in 2024, respectively, were primarily due to lower research, laboratory, and clinical development costs related to the portfolio prioritization announced in the fourth quarter of 2024, lower personnel-related, costs, including non-cash stock-based compensation, a decrease in facility and other allocated costs primarily due to the portfolio prioritization announced in the fourth quarter of 2024, and lower third-party manufacturing costs. Research and development expenses include non-cash stock-based compensation of $4.2 million and $8.8 million, respectively, for the three and six months ended June 30, 2025 and $7.1 million and $13.0 million for the same periods in 2024.
Research and Development Related Success Payments and Contingent Consideration: For the three and six months ended June 30, 2025, Sana recognized non-cash expenses of $10.3 million and $12.2 million, respectively, compared to a non-cash gain of $27.9 million and a non-cash expense of $10.1 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 six months ended June 30, 2025, inclusive of non-cash expenses, were $10.3 million and $21.8 million, respectively, compared to $16.4 million and $32.7 million for the same periods in 2024. The decreases of $6.1 million and $10.9 million for the three and six months ended June 30, 2025, respectively, compared to the same periods in 2024 were 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 $2.4 million and $4.8 million for the three and six months ended June 30, 2025, respectively, compared to $4.3 million and $7.5 million for the same periods in 2024.
Impairment of Long-Lived Assets: For the three and six months ended June 30, 2025, non-cash impairment of long-lived assets was $44.6 million, compared to zero for the same periods in 2024. The non-cash impairment 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 our near-term manufacturing needs, we expect to use CDMOs to meet our manufacturing needs at present and have suspended further build-out of our internal manufacturing capabilities.
Net Loss: Net loss for the three and six months ended June 30, 2025 was $93.8 million, or $0.39 per share, and $143.2 million, or $0.60 per share, respectively, compared to $50.3 million, or $0.21 per share, and $157.8 million, or $0.70 per share, for the same periods in 2024.

Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the six months ended June 30, 2025 was $79.0 million compared to $104.6 million for the same period in 2024. Non-GAAP operating cash burn is the decrease in cash, cash equivalents, and marketable securities, excluding cash inflows from financing activities, costs related to the portfolio prioritization in the fourth quarter of 2024 that were paid in 2025, and the purchase of property and equipment.
Non-GAAP Net Loss: Non-GAAP net loss for the three and six months ended June 30, 2025 was $38.9 million, or $0.16 per share, and $86.4 million, or $0.36 per share, respectively, compared to $74.2 million, or $0.32 per share, and $143.6 million, or $0.64 per share, for the same periods in 2024. Non-GAAP net loss excludes non-cash expenses and gains related to the change in the estimated fair value of contingent consideration and success payment liabilities, and non-cash impairment losses recorded in 2025.

On August 11, 2025 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases, reported an update on the company’s development pipeline and reported financial results for the second quarter ended June 30, 2025 (Press release, Quince Therapeutics, AUG 11, 2025, View Source [SID1234655075]).

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

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

                  Schedule Your 30 min Free Demo!

Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, said, "We achieved many critical milestones over the last quarter that significantly advance our research programs and strengthen our business model. Specifically, we completed enrollment in our pivotal Phase 3 NEAT clinical trial, secured additional financing to extend our operating runway sufficiently beyond topline results, and solidified our commercial development planning through our strategic partnership with Option Care Health. Quince remains confident in our ability to deliver topline results in the first quarter of 2026 and subsequent NDA submission in the second half of 2026, assuming positive study results."

Pivotal Phase 3 NEAT Clinical Trial

Quince completed enrollment in its pivotal Phase 3 NEAT (Neurological Effects of eDSP on Subjects with A-T; NCT06193200/IEDAT-04-2022) clinical trial with a total of 105 participants, including 83 participants in the six to nine year-old primary analysis population and 22 participants aged 10 years and older.
Quince expects to report topline results from its Phase 3 NEAT clinical trial in the first quarter of 2026.
Concluding the NEAT study with 83 enrolled participants in the six to nine year-old primary analysis population reflects powering of approximately 90% to determine statistical significance of the primary endpoint.
All 50 NEAT participants to date have elected to transition to the open label extension (OLE) study (NCT06664853/IEDAT-04-2022). Participants who complete the full treatment period, complete study assessments, and provide informed consent are eligible to transition to the OLE study.
The Phase 3 NEAT clinical trial is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA).
Assuming positive study results, the company plans to submit a New Drug Application (NDA) to the FDA in the second half of 2026.
Quince was granted FDA Fast Track designation for the company’s eDSP System for the treatment of patients with A-T based on the potential to address a high unmet medical need.
NEAT is an international, multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the neurological effects of Quince’s lead asset, eDSP (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells; previously referred to as EryDex) in patients with A-T.
Participants are randomized (1:1) between eDSP or placebo and treatment consists of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last efficacy visit using the Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo.
Pipeline and Corporate Updates

Announced a strategic relationship with Option Care Health, Inc. (Nasdaq: OPCH), the nation’s largest independent provider of home and ambulatory infusion services, to support the commercial development and efficient launch of Quince’s lead asset, eDSP, in the U.S. The strategic relationship will leverage Option Care’s robust network of specialty pharmacy and ambulatory infusion suites to provide for the administration of eDSP in an effective and efficient way while delivering this innovative treatment to patients with greater geographic flexibility.
Closed a private placement of common stock and accompanying warrants in June 2025 led by healthcare-focused institutional investor Nantahala Capital with participation from existing Quince stockholders including ADAR1 Capital Management, Legend Capital Partners, and Lagfin S.C.A., new stockholder Second Line Capital, along with members of Quince’s senior management. Priced at a more than a 10% premium to the market price of Quince’s common stock, the financing resulted in approximately $11.5 million in upfront proceeds and potential additional proceeds of up to $10.4 million, if the accompanying warrants are exercised in full, before deducting placement agent fees and other private placement expenses.
Finalized Phase 2 clinical trial study designs to evaluate eDSP for the potential treatment of patients with Duchenne muscular dystrophy (DMD), the company’s second targeted indication for its lead asset, eDSP. Quince plans to prioritize capital efficient study approaches, including potential investigator-initiated trials (IITs), to advance the evaluation of DMD as a second targeted eDSP indication.
Initiated Study #3 in the company’s European Union pediatric investigational plan (PIP) – named the Pediatric Encapsulated Dexamethasone Sodium Phosphate (PeD) study – to evaluate the safety and pharmacokinetics of eDSP in younger patients with A-T who weigh between nine and 15 kilograms.
Participated at the 2025 A-T Clinical Research Conference organized by the A-T Society, a leading A-T patient advocacy group based in the United Kingdom, where key opinion leaders (KOLs) presented post hoc data analyses from the company’s prior Phase 3 ATTeST clinical trial, in addition to Quince management providing an overview of the Phase 3 NEAT clinical trial.
Appointed Dr. Hassan Abolhassani, Assistant Professor of Clinical Immunology and Research Specialists in the Department of Medical Biochemistry and Biophysics at the Karolinska Institutet in Stockholm, Sweden, to the company’s Scientific Advisory Board (SAB). Dr. Abolhassani becomes the ninth member to join Quince’s SAB, which is comprised of leading experts in A-T, biochemistry, neurology, immunology, genetic, hematology, pharmacology, and clinical practice.
Second Quarter 2025 Financial Results

Reported cash, cash equivalents, and short-term investments of $34.7 million for the second quarter ended June 30, 2025. Quince expects its existing cash runway to be sufficient to fund the company’s capital efficient development plan through Phase 3 NEAT topline results into the second quarter of 2026. If warrants related to the company’s recent financing are exercised in full for cash, Quince’s cash runway would extend into the second half of 2026.
Reported research and development (R&D) expenses of $6.6 million for the second quarter ended June 30, 2025. R&D expenses primarily included costs related to ongoing Phase 3 NEAT clinical trial activities and related manufacturing costs.
Reported general and administrative (G&A) expenses of $3.3 million for the second quarter ended June 30, 2025. G&A expenses primarily included personnel-related and stock-based compensation expenses, commercial planning and new product planning expenses, and other professional administrative costs.
Reported a net loss of $16.1 million, or a net loss of $0.34 per basic and diluted share, for the second quarter ended June 30, 2025. Weighted average shares outstanding for the year were 46.7 million.
Reported net cash used in operating activities of $21.0 million for the six months ended June 30, 2025. Cash used in operating activities was primarily due to net loss of $31.1 million for the period, adjusted for $9.9 million of non-cash items, including $4.5 million change in the fair value of warrants, $2.7 million in stock-based compensation, $2.5 million change in the fair value of contingent consideration liabilities, $0.8 million change in the fair value of the European Investment Bank loan, and a net decrease in operating assets of $0.5 million, offset by a net increase in accounts payable, and accrued expenses, and other current liabilities of $0.3 million.