Arrowhead Pharmaceuticals Reports Fiscal 2026 First Quarter Results

On February 5, 2026 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2026 first quarter ended December 31, 2025. The Company is hosting a conference call today, February 5, 2026, at 4:30 p.m. ET to discuss the results.

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 had another quarter of strong execution across all areas of our business and we think Arrowhead is extremely well positioned to build on this progress throughout 2026 and beyond," said Christopher Anzalone, Ph.D., President and CEO at Arrowhead. "In fact, the recent months have included some of the more significant achievements in our Company’s history. We received regulatory approval for REDEMPLO in familial chylomicronemia syndrome in three different countries and launched our first commercial product in the U.S.; we continued to grow our cardiometabolic portfolio; we had encouraging early results from our obesity programs; we advanced our TRiM platform and CNS pipeline; and, lastly, we meaningfully improved our financial position to advance these and other programs forward."

Webcast and Conference Call and Details

Investors may access a live audio webcast on the Events and Presentations page under the Investors section of the Arrowhead website. A replay of the webcast will be available approximately two hours after the conclusion of the call.

For analysts that wish to participate in the conference call, please register at View Source Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call.

Key Commercial Events

Announced that on November 18, 2025, the U.S. FDA approved REDEMPLO (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS);
FCS is a severe, rare disease, with an estimated 6,500 people in the U.S. living with genetic or clinical FCS, characterized by triglyceride levels that can be 10 to 100 times higher than normal leading to a substantially higher risk of developing acute, recurrent, and potentially fatal pancreatitis;
This is Arrowhead’s first FDA-approved medicine, marking a major milestone for the company as it transitions into commercial-stage;
Launched REDEMPLO independently in the U.S. with the One-REDEMPLO pricing model that creates a consistent price across current and potential future indications. Initial trends in prescriptions, payor reviews and reimbursement, and early shipments have been encouraging and include the following, to date:
Over 100 prescriptions for REDEMPLO have been received from a diverse prescriber base, with geographically balanced uptake across the U.S.;
Early patient starts fall into three categories: patients transitioning from our Expanded Access Program, patients naïve to the APOC3 class, and patients switching from olezarsen;
Patients receiving REDEMPLO include both clinically diagnosed and genetically confirmed FCS, with the majority not required to submit genetic testing to gain access;
Launched Rely On REDEMPLO, a patient support program providing support services and resources for patients at each stage of the treatment journey with REDEMPLO, including financial assistance options for eligible patients;
Announced that the Chinese National Medical Products Administration (NMPA) has approved REDEMPLO (plozasiran) for the reduction of triglyceride levels in adult patients with familial chylomicronemia syndrome. REDEMPLO will be marketed in Greater China by Sanofi under an agreement between Sanofi and Arrowhead;
Announced that Health Canada has issued a Notice of Compliance (NOC) authorizing REDEMPLO (plozasiran) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome for whom standard triglyceride lowering therapies have been inadequate. REDEMPLO will be available later this year in Canada and the company anticipates it will be marketed independently by Arrowhead;
Key R&D Events

Initiated and dosed the first subjects in a Phase 1/2a clinical trial of ARO-DIMER-PA, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia. ARO-DIMER-PA is designed to silence expression of the proprotein convertase subtilisin kexin 9 (PCSK9) and apolipoprotein C3 (APOC3) genes. This represents an important step forward for the RNAi field as it is the first dual-function clinical candidate to target two genes simultaneously in one molecule;
Announced interim clinical data on our RNAi-based obesity candidates, ARO-INHBE and ARO-ALK7, showing weight loss in obese patients with diabetes and improved measures of body composition;
ARO-INHBE in combination with tirzepatide achieved -9.4% weight loss at week 16 in obese patients with type 2 diabetes mellitus, demonstrating an approximately two-fold improvement versus -4.8% on tirzepatide alone;
ARO-INHBE drove robust fat reduction including -23.2% visceral fat, -15.4% total fat, and -76.7% liver fat reduction, representing an approximately three-fold improvement in all these measures versus tirzepatide alone in obese diabetic patients;
ARO-ALK7 is the first RNAi-therapeutic to show knockdown in humans of an adipocyte expressed gene and achieved a mean reduction of -88% in ALK7 mRNA with a maximum reduction of -94%;
ARO-ALK7 monotherapy achieved a -14.1% (single dose, week 8) placebo adjusted visceral fat reduction;
Initiated and dosed the first subjects in a Phase 1/2a clinical trial of ARO-MAPT, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies including Alzheimer’s disease, a progressive neurodegenerative disease characterized by cognitive and functional decline. ARO-MAPT is Arrowhead’s first investigational RNAi-based therapy to utilize a new proprietary delivery system which, in preclinical studies, has achieved blood-brain-barrier penetration and deep knockdown of target genes across the central nervous system (CNS), including deep brain regions, after subcutaneous injection;
Announced that the U.S. FDA has granted Breakthrough Therapy designation to investigational plozasiran as an adjunct to diet to reduce triglyceride (TG) levels in adults with severe hypertriglyceridemia (SHTG) (TG levels greater than or equal to 500 mg/dL);
Key Corporate Events

Closed two concurrent public offerings with gross proceeds totaling $930,000,000 and consisting of (i) 0.00% convertible senior notes due 2032 (the "notes") and (ii) shares of common stock, at a public offering price of $64.50 per share (or, in lieu of shares of common stock to certain investors, pre-funded warrants);
Triggered a $200.0 million milestone payment from Sarepta Therapeutics, Inc., which was earned on November 20, 2025, when the Company reached the second of two prespecified enrollment targets and subsequent authorization to dose escalate in a Phase 1/2 clinical study of SRP-1003 (formerly ARO-DM1), an investigational RNAi therapeutic for the treatment of type 1 myotonic dystrophy (DM1);
Announced a global licensing and collaboration agreement with Novartis, which closed on October 17, 2025, for ARO-SNCA, Arrowhead’s preclinical stage siRNA therapy against alpha-synuclein for the treatment of synucleinopathies, such as Parkinson’s Disease, and for other additional collaboration targets that will utilize Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform. Financial terms of the agreement include:
Arrowhead received a $200 million upfront payment from Novartis. Arrowhead is also eligible to receive development, regulatory, and sales milestone payments of up to $2 billion. Arrowhead is further eligible to receive tiered royalties on commercial sales up to the low double digits.
Selected Fiscal 2026 First Quarter Financial Results

ARROWHEAD PHARMACEUTICALS, INC.

CONSOLIDATED CONDENSED FINANCIAL INFORMATION

(in thousands, except per share amounts)

Three months Ended December 31,

OPERATING SUMMARY

2025

2024

Revenue

$

264,033

$

2,500

Operating Expenses:

Research and development

177,203

137,002

General and administrative expenses

46,021

26,910

Total operating expenses

223,224

163,912

Operating income (loss)

40,809

(161,412

)

Total other expense

(12,538

)

(13,703

)

Income (loss) before income tax expense and noncontrolling interest

28,271

(175,115

)

Income tax expense

29

103

Net income (loss) including noncontrolling interest

28,242

(175,218

)

Net loss attributable to noncontrolling interest, net of tax

(2,569

)

(2,133

)

Net income (loss) attributable to Arrowhead Pharmaceuticals, Inc.

$

30,811

$

(173,085

)

Net income (loss) per share attributable to Arrowhead Pharmaceuticals, Inc. – Diluted

$

0.22

$

(1.39

)

Weighted-average shares used in calculating – Diluted

140,706

124,848

December 31,
2025

September 30,
2025

FINANCIAL POSITION SUMMARY


(unaudited)

Cash, cash equivalents and restricted cash


$

201,642

$

226,548

Available-for-sale securities, at fair value and short-term investments

714,967

692,818

Total cash resources (Cash, cash equivalents and restricted cash and Available-for-sale securities, at fair value and short-term investments)

916,609

919,366

Other current and long-term assets

687,572

465,929

Total Assets


$

1,604,181

$

1,385,295

Liability related to the sale of future royalties


$

374,997

$

367,397

Credit Facility

203,108

254,883

Deferred revenue

165,758

2,399

Other liabilities

297,621

257,200

Total Liabilities


$

1,041,484

$

881,879

Total Arrowhead Pharmaceuticals, Inc. Stockholders’ Equity

568,422

466,052

Noncontrolling Interest

(5,725

)

37,364

Total Noncontrolling Interest and Stockholders’ Equity


$

562,697

$

503,416

Total Liabilities, Noncontrolling Interest and Stockholders’ Equity


$

1,604,181

$

1,385,295

Shares Outstanding


137,391

135,702

About REDEMPLO (plozasiran)

REDEMPLO (plozasiran) is approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with Familial Chylomicronemia Syndrome (FCS). REDEMPLO is an siRNA therapeutic designed to suppress the production of apoC-III, a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting apoC-III with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is the first and only siRNA FDA-approved treatment studied in both genetically confirmed and clinically diagnosed patients living with FCS.

For more information about REDEMPLO, visit Our Medicines.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

None.

ADVERSE REACTIONS

Most common adverse reactions in REDEMPLO treated patients (incidence ≥10% of patients treated with REDEMPLO and >5% more frequently than with placebo) are hyperglycemia, headache, nausea, and injection site reaction.

Please see full Prescribing Information for REDEMPLO.

(Press release, Arrowhead Pharmaceuticals, FEB 5, 2026, View Source [SID1234662514])

TransCode Therapeutics and Quantum Leap Announce Submission of IND Amendment for Phase 2a Clinical Trial with TTX-MC138

On February 5, 2026 TransCode Therapeutics, Inc. (NASDAQ: RNAZ) (TransCode), a clinical stage company pioneering immuno-oncology and RNA therapeutics for the treatment of high risk and advanced cancers, in collaboration with Quantum Leap Healthcare Collaborative (Quantum Leap), reported the submission to the U.S. Food and Drug Administration (FDA) of an Investigational New Drug (IND) application amendment for a planned Phase 2a clinical trial with TransCode’s lead therapeutic candidate, TTX-MC138. The study will be conducted by Quantum Leap within their PRE-I-SPY program, a leading platform for innovative oncology clinical trials, and represents the program’s first expansion into colorectal cancer.

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!

As part of the PRE-I-SPY platform, the TTX-MC138 Phase 2a dose-expansion portion of the trial will enroll up to 45 patients with colorectal cancer who have completed standard curative-intent therapy and have positive markers for circulating tumor DNA (ctDNA). Recent studies have demonstrated that ctDNA is a prognostic marker of cancer recurrence and may be indicative of the presence of minimal residual disease (MRD) that could be amenable to early interventions. The Phase 2a trial is planned to begin in the first half of 2026 and will be led by Principal Investigator Dr. Paula Pohlmann of MD Anderson Cancer Center. This clinical trial aims to evaluate the biological and clinical activity of TTX-MC138 in the MRD setting, where we believe the therapeutic intervention may have the greatest opportunity to improve long-term outcomes.

"This IND submission marks a pivotal step in TransCode’s clinical development program, positioning TTX-MC138 where its mechanism of action has the potential to deliver meaningful benefit to patients," said Sue Duggan, TransCode’s Senior VP, Operations. "We are pleased to partner with Quantum Leap’s PRE-I-SPY program to evaluate TTX-MC138 and to support the expansion of this platform into new indications such as colorectal cancer," added Duggan.

The clinical trial will be performed at several clinical sites of the PRE-I-SPY Platform Network, many of which are members of the National Cancer Center Network. Additionally, the program is focused on partnering with the Colorectal Cancer Alliance, a leading advocacy organization. Information on the PRE-I-SPY program and the Phase 2a trial can be found at clinicaltrials.gov (NCT05868226).

(Press release, TransCode Therapeutics, FEB 5, 2026, View Source [SID1234662512])

Syndax Announces Participation at the 2026 Guggenheim Emerging Outlook: Biotech Summit

On February 5, 2026 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported that Michael A. Metzger, Chief Executive Officer, will participate in a fireside chat at the 2026 Guggenheim Emerging Outlook: Biotech Summit on Thursday, February 12, 2026, at 12:30 p.m. ET.

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 webcast of the fireside chat will be available in the Investor section of the Company’s website at www.syndax.com, where a replay will also be available for a limited time.

(Press release, Syndax, FEB 5, 2026, View Source [SID1234662511])

ORIC® Pharmaceuticals to Participate in Upcoming Investor Conferences

On February 5, 2026 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported that management will be participating in the following investor conferences in February:

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!

Guggenheim Emerging Outlook: Biotech Summit – Participating in a fireside chat on Thursday, February 12, 2026, at 10:30 a.m. ET. Management will also be participating in one-on-one meetings.

Citi’s 2026 Virtual Oncology Leadership Summit – Participating in a fireside chat on Wednesday, February 18, 2026, at 2:30 p.m. ET. Management will also be participating in one-on-one meetings.

Oppenheimer 36th Annual Healthcare Life Sciences – Participating in a fireside chat on Wednesday, February 25, 2026, at 12:00 p.m. ET. Management will also be participating in one-on-one meetings.

Webcasts of the fireside chat discussions will be available through the investor section of the company’s website at www.oricpharma.com. Replays of the webcasts will be available for 90 days following the event.

(Press release, ORIC Pharmaceuticals, FEB 5, 2026, View Source [SID1234662510])

Orca Bio Presents New Data at the 2026 Tandem Meetings of ASTCT® and CIBMTR® Reinforcing Orca-T® as a Durable, High-Precision Cell Therapy for Hematological Malignancies

On February 5, 2026 Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, reported new clinical data presented at the 2026 Tandem Meetings of ASTCT and CIBMTR from February 4-7 in Salt Lake City.

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!

Orca-T with Reduced Intensity Conditioning

"Patients undergoing reduced intensity conditioning allogeneic stem cell transplantation often face a tradeoff between tolerability and long-term disease control," said Caspian Oliai, MD, medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center, and primary investigator on the SERENE-T Phase 2 study. "The clinical evidence being generated today, which suggests Orca-T may improve key outcomes by reducing GVHD without increasing infection risk or relapse rate, provides a strong foundation for our ongoing evaluation of Orca-T in this setting. The dosing of the first patients in the Phase 2 SERENE‑T study further strengthens this initial momentum, representing a meaningful step forward in expanding the investigation of Orca‑T for patients undergoing reduced intensity conditioning."

SERENE-T (NCT07216443) is a new multicenter, open-label Phase 2 trial evaluating the safety, tolerability and efficacy of Orca-T, Orca Bio’s lead investigational allogeneic T-cell immunotherapy, in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) undergoing reduced intensity conditioning (RIC) or non-myeloablative conditioning (NMA). The first patients were treated this year at Vanderbilt University, UCLA and Oregon Health & Science University (OHSU) – Knight Cancer Institute. The study continues to enroll patients with plans to open at additional transplant centers across the U.S.

A new analysis compared outcomes from the single-center, open-label Phase 1 investigator-sponsored trial evaluating Orca-T versus a historical cohort of patients from the CIBMTR registry who received a conventional allogeneic hematopoietic stem cell transplant (alloHSCT) with post-transplant cyclophosphamide (PTCy). All patients were aged 60-75 (median 68 years), had a 7/8 or 8/8 human leukocyte antigen (HLA)-matched donor, and were given a RIC for the treatment of AML, acute lymphoblastic leukemia (ALL), MDS or myeloproliferative neoplasm (MPN).

Patients receiving Orca-T (n=53) compared with PTCy (n=587) demonstrated improved:

Overall survival at one year (OS; 88% vs 72%) and two years (84% vs 61%)
Relapse-free survival at one year (RFS; 82% vs 61%) and two years (79% vs 53%)
Graft-versus-host-disease relapse-free survival at one year (GRFS; 72% vs 54%) and two years (72% vs 45%)
Relapse rates at one and two years (9.7% vs 23%, 9.7% vs 30%) and non-relapse mortality at one and two years (NRM; 8% vs 16%, 12% vs 17%).
At one year, rates of Grade 3-4 acute and moderate-to-severe chronic graft versus host disease (aGVHD, cGVHD) were 0% and 10% with Orca-T, respectively, compared to 6% and 10% with PTCy. At two years, rates of cGVHD were 10% with Orca-T and 12% with PTCy.

Orca-T Versus PTCy in Patients with Myelodysplastic Syndromes

A post-hoc analysis of patients aged 18-65 with MDS compared pooled results from the Phase 3 Precision-T study and the Phase 1b study of Orca-T to a historical cohort from the CIBMTR registry of patients who received a conventional alloHSCT and PTCy in the myeloablative conditioning (MAC) HLA-matched setting. Patients with MDS who were treated with Orca-T (n=25) demonstrated higher one-, two- and three-year OS of 100% compared to the PTCy cohort (n=95) with 80%, 70% and 62%, respectively. At one year, the Orca-T arm showed RFS of 95% versus 64% with PTCy and NRM of 0% versus 9.9%, respectively. Notably, these trends were observed across subgroups including age and donor type.

A similar analysis was conducted across multiple hematologic malignancies, including MDS, AML and ALL with consistent results. In this post-hoc analysis, Orca-T (n=164) demonstrated higher OS compared to PTCy (n=380) at one, two and three years (94% vs 82%, 85% vs 73% and 82% vs 65%, respectively). At one year, RFS was 78% with Orca-T compared with 70% with PTCy, while NRM was 2.7% versus 7.7%, respectively. These findings were consistent with the results observed in a subgroup of patients over 50 years of age.

Reliable Manufacturing and Nationwide Distribution of Orca-T

A manufacturing and distribution analysis from the Phase 3 and Phase 1b Orca-T studies conducted between December 2019 and September 2024 reported on the production of 243 clinical cell therapies, including 215 from single-day and 28 from two-day collections. Overall, 100% of products were delivered to transplant centers across the U.S. within 70 hours, with 99% infused within 72 hours. Product quality was consistent across all three Orca-T components: hematopoietic stem cells, regulatory T-cells and conventional T-cells.

These results demonstrate the feasibility of reliably manufacturing and distributing Orca-T at scale while maintaining high product purity within a controlled logistics framework, supporting multicenter clinical studies and potential future commercial application.

"These data continue to reinforce the strength of Orca-T across both clinical outcomes and operational execution," said Nate Fernhoff, Ph.D., co-founder and chief executive officer at Orca Bio. "The consistency of these results, along with our ability to reliably manufacture and deliver Orca-T across the U.S., highlights the potential of this therapy to make a meaningful difference for patients with hematologic malignancies. As we move toward a potential launch later this year, we remain focused on executing with the same level of commitment and rigor to support patients, clinicians and transplant centers."

Orca-Q for Patients with Haploidentical Donors

New findings from the ongoing Phase 1 study of Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy, evaluated 39 patients with AML, ALL or MDS and a haploidentical donor who received MAC with Bu/Flu/Thiotepa (BFT), TBI/Flu/Thio (TFT) or TBI/Flu. All patients engrafted by Day +19 (median 11 days) and demonstrated encouraging rates of OS at one, two and three years (80%, 77% and 77%, respectively). Patients demonstrated RFS of 77% and GRFS of 72%, with low incidences of Grade 3-4 aGVHD at Day +180 and moderate-to-severe cGVHD at one year (8.1% and 0%, respectively). Outcomes were further improved in the TFT subgroup (n=14) across OS (85%), RFS (85%), GRFS (85%), aGVHD (0%) and cGVHD (0%).

About Orca-T
Orca-T is an investigational allogeneic T-cell immunotherapy under evaluation for the treatment of multiple hematologic malignancies including acute leukemias and myelodysplastic syndromes. Orca-T is composed of highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the U.S. Food and Drug Administration (FDA). The Biologics License Application (BLA) for Orca-T is currently under Priority Review with the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026.

About Orca-Q
Orca-Q is Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies, including in patients with haploidentical and mismatched donors. Orca-Q is a proprietary composition of stem cells combined with specific T-cell subsets derived from healthy donors and engineered by Orca Bio’s high-precision platform.

(Press release, Orca Bio, FEB 5, 2026, View Source;utm_medium=rss&utm_campaign=orca-bio-presents-new-data-at-the-2026-tandem-meetings-of-astct-and-cibmtr-reinforcing-orca-t-as-a-durable-high-precision-cell-therapy-for-hematological-malignancies [SID1234662509])