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])

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;

o

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

o

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

o

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.

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!

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 3, 2026 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported an update on the potential spin-off of its biopharma operations and reported financial results for the fourth quarter and year ended Dec. 31, 2025.

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 approaching a defining inflection point for Anaptys, as we plan to spin-off in Q2 2026 our wholly owned biopharma portfolio into a public company, to be called First Tracks Biotherapeutics, to unlock and amplify value for investors across two distinct sets of assets," said Daniel Faga, president and chief executive officer of Anaptys. "In our royalty portfolio, Jemperli exited Q4 2025 on a ~$1.4 billion annualized run rate, reinforcing GSK’s peak sales guidance of far more than $2.7 billion2 in monotherapy indications. At the same time, our biopharma portfolio is advancing multiple attractive, high-potential assets, including ANB033, which has pipeline-in-a-product potential, initially in a Phase 1b trial for both celiac disease and eosinophilic esophagitis."

INTENT TO SEPARATE BUSINESS

Intention to separate biopharma operations from substantial royalty assets on track for Q2 2026, potentially as early as late-April
Designed to unlock potential value by creating two independent, publicly traded companies with different business objectives and opportunities
The royalty management company will initially retain the name AnaptysBio (Nasdaq: ANAB) and will manage the financial collaborations from Jemperli with GSK and imsidolimab with Vanda, with a focus on protecting and returning their value to shareholders
While specific decisions regarding board composition, leadership and financial operations will be disclosed at a later time, Daniel Faga is anticipated to be the initial CEO
First Tracks Biotherapeutics, Inc. (Nasdaq: TRAX) (formerly referred to as Biopharma Co), will be a public company focused on the development and potential commercialization of innovative immunology therapeutics for autoimmune and inflammatory diseases, including ANB033, rosnilimab and ANB101
Form 10 registration statement has been publicly filed in connection with the planned spin-off
Initial Board of Directors for First Tracks Biotherapeutics is expected to include certain current members of Anaptys’ Board: Daniel Faga, Dennis Fenton, Ph.D., John Orwin (Chairman), John Schmid, Magda Marquet, Ph.D., Rita Jain, M.D., and Tony Ware, M.D.
Initial executive leadership team for First Tracks Biotherapeutics will include Daniel Faga, CEO, Paul Lizzul, CMO and Ben Stone, CBO. Additional executives will be disclosed at a later time.
Upon completion of the spin-off, First Tracks Biotherapeutics will launch with adequate capital to fund operations through significant potential product milestones

AnaptysBio (formerly referred to as "Royalty Management Co")

GSK Jemperli Financial Collaboration

GSK announced strong commercial performance for Jemperli ($343 million/£261 million in Q4 2025 sales; $1.128 billion/£861 million in YTD 2025 sales) with >13% USD and GBP quarter-over-quarter growth1
Implies a ~$1.4 billion annualized run rate
In Dec. 2025, Anaptys received a one-time $75 million commercial sales milestone from GSK when Jemperli achieved $1 billion in worldwide net sales in Nov. 2025
Anaptys expects to achieve >$390 million in annualized Jemperli royalties payable to Anaptys at GSK’s peak sales guidance of >$2.7 billion2 as early as 2029
Anaptys estimates Sagard will have accrued ~$250 million in royalties and sales milestones through year-end 2025 and anticipates full paydown of $600 million non-recourse debt monetization by the end of Q2 20273
Substantial GSK investment in additional monotherapy and potential combination trials for Jemperli, including:
AZUR-1 – pivotal Phase 2 – dostarlimab monotherapy in untreated stage II/III dMMR/MSI-H locally advanced rectal cancer
Top-line data expected in 2026; U.S. FDA Breakthrough Therapy Designation
Received an FDA Commissioner’s National Priority Voucher (CNPV) in Nov. 2025 allowing for only a one to two-month BLA review timeline for US FDA approval
AZUR-2 – pivotal Phase 3 – dostarlimab versus standard of care in untreated TN40 or stage III dMMR/ MSI-H resectable colon cancer
Top-line data expected in 2028
AZUR-4 – Phase 2 – dostarlimab plus chemotherapy versus standard of care (chemotherapy) in untreated stage III MMRp/MSS resectable colon cancer
Top-line data expected in Q4 2026
JADE – pivotal Phase 3 – dostarlimab monotherapy versus placebo in locally advanced unresected head and neck squamous cell carcinoma (PD-L1 hiPghD-L1 CPS≥1) post chemoradiation
Top-line data expected in 2028

Vanda Imsidolimab Financial Collaboration

FDA accepted the BLA filing for imsidolimab in generalized pustular psoriasis (GPP) in Feb. 2026 with a target action date of Dec. 12, 2026

First Tracks Biotherapeutics (formerly referred to as "Biopharma Co")

ANB033 (CD122 antagonist)

Phase 1b trial in celiac disease ongoing
60-patient trial assessing one dose level of subcutaneously administered ANB033 vs. placebo (randomized 1:1) across two different cohorts
Cohort 1 (n=30) is a gluten-challenge study to assess the prevention of mucosal damage
Patients enrolled have a Vh:Cd ratio of >2.0 are treated with ANB033 or placebo for 4 weeks, and after are administered a daily 6-gram gluten challenge at Week 4 for 14 days, and are assessed at Week 6 via biopsy
Cohort 2 (n=30) is a study to assess the possibility of mucosal healing in the likely commercial population
Patients enrolled have a Vh:Cd ratio of <2.0 are treated with ANB033 or placebo for 4 weeks and are assed at Week 12 via biopsy
Top-line Phase 1b data for both cohorts anticipated in Q4 2026
Phase 1b trial in eosinophilic esophagitis initiated in Q1 2026
50-patient cohort assessing one dose level of subcutaneously administered ANB033 vs. placebo (randomized 1:1)
Top-line Phase 1b data anticipated in 2027
Rosnilimab (Pathogenic T Cell Depleter)

Presented Phase 2b data for rosnilimab, a pathogenic T cell depleter, in rheumatoid arthritis as a late-breaking oral presentation at American College of Rheumatology (ACR) Convergence 2025
Presentation available on the Anaptys website here
Anticipate providing an update on advancement of rosnilimab in RA, which would be funded by strategic or other outside sources of capital, in Q2 2026
ANB101 (BDCA2 modulator)

Phase 1a trial in healthy volunteers ongoing
To date, ANB101’s preclinical and Phase 1a data have suggested it is a more potent antibody with longer half-life resulting in deeper and more durable PD effect of pDC depletion vs. Biogen’s litifilimab, a competing BDCA2 modulator
FINANCIAL UPDATES

Cash Position and Stock Repurchase Program

Cash and investments of $311.6 million as of Dec. 31, 2025
Company has repurchased a total of 3,444,079 shares of common stock (11.2% shares outstanding) with $68.6 million as of Dec. 31, 2025, from its $175.0 million Stock Repurchase Program, which expires March 31, 2026
Fourth Quarter and Full Year 2025 Financial Results

Cash, cash equivalents and investments totaled $311.6 million as of Dec. 31, 2025, compared to $420.8 million as of Dec. 31, 2024, for a decrease of $109.2 million due primarily to $130.6 million used for operating activities and $68.6 million in shares repurchased offset by $75.0 million received from GSK for Jemperli total sales for 2025 exceeding $1.0 billion and $15.0 million received from Vanda Pharmaceuticals for the license of imsidolimab.
Collaboration revenue was $108.2 million and $234.6 million for the three and twelve months ended Dec. 31, 2025, compared to $43.1 million and $91.3 million for the three and twelve months ended Dec. 31, 2024. The increase was due primarily to Jemperli total sales for 2025 exceeding $1.0 billion which earned one-time $50 million and $75 million commercial sales milestones under our license agreement with GSK, Jemperli royalties increased 89% from $17.3 million to $32.7 million and 103% from $47.4 million to $96.0 million for the three and twelve months ended Dec. 31, 2025, and $9.7 million in revenue recognized for the Vanda license agreement.
Research and development expenses were $25.6 million and $136.0 million for the three and twelve months ended Dec. 31, 2025, compared to $42.6 million and $163.8 million for the three and twelve months ended Dec. 31, 2024. The decrease for the three and twelve months ended Dec. 31, 2025, was primarily due to decreased development costs for ANB032, rosnilimab, and imsidolimab, offset by increased costs relating to the Phase 1 trials for ANB033 and ANB101. The R&D non-cash, stock-based compensation expense was $3.8 million and $17.1 million for the three and twelve months ended Dec. 31, 2025, as compared to $3.9 million and $14.8 million in the same period in 2024.
General and administrative expenses were $15.8 million and $50.7 million for the three and twelve months ended Dec. 31, 2025, compared to $10.2 million and $42.4 million for the three and twelve months ended Dec. 31, 2024. The increase was due primarily to legal costs including the separation of the company and transaction costs associated with the Vanda Pharmaceuticals license agreement. The G&A non-cash, stock-based compensation expense was $4.7 million and $18.9 million for the three and twelve months ended Dec. 31, 2025, as compared to $4.3 million and $19.2 million in the same period in 2024.
Net income was $49.6 million for the three months ended Dec. 31, 2025, or a net income per share of $1.79 and a net loss of $13.2 million for the twelve months ended Dec. 31, 2025, or a net loss per share of $0.46, compared to a net loss of $21.8 million and $145.2 million for the three and twelve months ended Dec. 31, 2024, or a net loss per share of $0.72 and $5.12.

(Press release, AnaptysBio, MAR 3, 2026, View Source [SID1234663218])