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.

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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])

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.

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"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])

Akeso Receives Fifth Breakthrough Therapy Designation from NMPA for Ivonescimab in First-Line Treatment of Advanced Biliary Tract Cancer

On February 5, 2026 Akeso, Inc. (9926.HK) reported that ivonescimab, its global first-in-class bispecific antibody targeting PD-1 and VEGF, has been granted its fifth Breakthrough Therapy Designation from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA). This latest designation applies to ivonescimab in combination with chemotherapy for the first-line treatment of advanced biliary tract cancer (BTC).

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This milestone represents the fifth BTD awarded to ivonescimab by the NMPA, following three prior designations in lung cancer indications and one for triple-negative breast cancer (TNBC). The repeated recognition highlights ivonescimab’s broad clinical potential across multiple high unmet need tumor types.

A randomized, controlled, multicenter, registrational Phase III clinical study (AK112-309/HARMONi-GI1) is evaluating ivonescimab plus chemotherapy versus durvalumab (a PD-L1 inhibitor) plus chemotherapy for first-line treatment of advanced BTC. Patient enrollment has been completed, and the BTD status for this indication underscores the promising clinical profile of ivonescimab. The BTD status is expected to accelerate both the ongoing clinical development and the regulatory review process in China.

Encouraging results from a Phase 1b/II study, presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual meeting, support the potential of the ivonescimab combination therapy as a superior first-line treatment for advanced BTC. In the study, ivonescimab plus chemotherapy achieved an Objective Response Rate (ORR) of 63.6% and a Disease Control Rate (DCR) of 100%. The ivonescimab regimen also demonstrated a median Progression-Free Survival (mPFS) of 8.5 months and a median Overall Survival (mOS) of 16.8 months.

These compelling Phase II results provide a robust foundation for the ongoing Phase III registrational trial and reinforce ivonescimab’s potential to address the significant unmet needs in advanced BTC, where current treatment options often yield limited durable responses.

(Press release, Akeso Biopharma, FEB 5, 2026, View Source [SID1234662516])

AIM ImmunoTech Reports Positive Year-End Interim Clinical Progress from Phase 2 Study Evaluating Ampligen® (rintatolimod) in Combination with AstraZeneca’s Imfinzi® (durvalumab) for the Treatment of Pancreatic Cancer

On February 5, 2026 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM" or the "Company") reported positive data in a year-end update from the ongoing Phase 2 clinical study evaluating AIM’s drug Ampligen (rintatolimod) combined with AstraZeneca’s anti-PD-L1 immune checkpoint inhibitor Imfinzi (durvalumab) in the treatment of metastatic pancreatic cancer patients with stable disease post-FOLFIRINOX standard of care (the "DURIPANC" study) (see: ClinicalTrials.gov NCT05927142). This is a follow-up Phase 2 to a 57-subject early access program ("EAP") of Ampligen as a monotherapy in late-stage pancreatic cancer, where Ampligen was associated with median survival of 19.7 months, which is an extension of median overall survival of 8.6 months when compared to the standard of care. The EAP subjects also reported improved quality of life.

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AIM CEO Thomas K. Equels states: "We know all too well that metastatic pancreatic cancer is a killer. Ampligen has the potential to be a gamechanger in the treatment of this highly lethal and unmet oncological need. Quality of life for pancreatic cancer patients is extremely painful and subject to co-morbidities due to the tumor-induced immune suppressive state. Additionally, other metastatic pancreatic cancer chemotherapies and immunotherapies typically have harsh side effects. However, Erasmus has informed us that the pancreatic cancer patients who received Ampligen have reported meaningful improvements in their quality of life. This data sharply focuses our aim on late-stage pancreatic cancers, which killed more than 100,000 people in the American and European Union markets and more than 450,000 people worldwide as recently as 2022. I do not believe there is any other therapeutic in this stage of the pipeline that is producing these types of survival results combined with improvement in quality of life."

The DURIPANC study is an investigator-initiated, exploratory, open-label, single-center study expected to enroll up to 25 subjects in the Phase 2 portion. The clinical trial is a joint collaboration between AIM, AstraZeneca and Erasmus Medical Center ("Erasmus MC") in the Netherlands. The primary objective of the study is the clinical benefit rate of the combination therapy. The secondary/exploratory objectives include assessing overall survival (OS) and progression-free survival (PFS); exploring immune-monitoring using available tissue biopsies and peripheral immune profiling; and assessing quality of life.

Eighteen patients have been enrolled in the study. Lead investigator Marjolein Y. V. Homs, MD, PhD, Department of Medical Oncology, Erasmus MC Cancer Institute, emphasized that the promising Progression-Free Survival and Overall Survival seen in Phase 1 of the study – which supported advancement to the ongoing Phase 2 portion of the study – continue to be seen and that enrollment is ongoing. Erasmus MC expects that detailed data will be published later this year.

According to Erasmus MC, there has also been no significant toxicity – an encouraging safety profile for a post-chemo setting – and Ampligen subjects are consistently reporting "high quality of life" during treatment.

See: DURIPANC, Year-End Interim Clinical Progress Update

Prof. Casper van Eijck, MD, PhD, of Erasmus MC, states: "Erasmus MC clinicians and researchers are seeing immune system changes that suggest a coordinated activation of innate and adaptive responses – or, to put it more simply, the combination of Ampligen and Durvalumab seems to be enhancing the body’s natural immune system. This perceived mechanism of action together with the clinical results supports continued investigation of this combination in post-FOLFIRINOX patients with pancreatic ductal adenocarcinoma."

Additionally, AIM has published on its website an updated corporate presentation that emphasizes the Company’s priority goal of a new drug approval for Ampligen in the treatment of pancreatic cancer. The presentation details AIM’s research and development work in pancreatic cancer; how Ampligen is believed to work in the treatment of pancreatic cancer; and why AIM believes that pancreatic cancer research and development holds the most potential for AIM’s stockholders. The largest mergers and acquisitions deals in the biotech space often involve oncology drugs in Phase 3 clinical trials or later in development, and so AIM believes that moving Ampligen toward – and ultimately into – a Phase 3 clinical trial has great financial potential for the Company and its stockholders.

See: Ampligen Breakthroughs in Treating Late-Stage Pancreatic Cancer: Corporate Presentation – February 2026

AIM’s intellectual property portfolio includes a U.S. patent for Ampligen as an oncology treatment in combination with anti-PD-L1 therapies, similar to that seen in the DURIPANC clinical trial combining Ampligen and AstraZeneca’s durvalumab; this patent extends protection to August 9, 2039. AIM has also been awarded orphan drug designations in pancreatic cancer by both the United States and the European Union, granting years of market exclusivity to AIM for Ampligen post-commercial approval.

Equels adds: "This patent protection and the orphan drug designations’ market exclusivity have the potential to create great value for our stockholders in this large-market unmet medical need."

(Press release, AIM ImmunoTech, FEB 5, 2026, View Source [SID1234662499])

K36 Therapeutics Completes Dosing of First Cohort in Phase 1 Clinical Trial of KTX-2001 in Prostate Cancer, Announces New CMO

On February 5, 2026 K36 Therapeutics, Inc. ("K36"), a privately held clinical-stage biotechnology company developing novel targeted therapies for cancers with high unmet medical need, reported completion of dosing in the first patient cohort of its Phase 1 clinical trial evaluating KTX-2001, a first-in-class, orally administered, selective NSD2 inhibitor, in patients with metastatic castration-resistant prostate cancer (mCRPC). This study marks the company’s second NSD2 inhibitor to enter the clinic.

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The Phase 1 clinical trial, STRIKE-001 (NCT07103018), is a multi-center, open-label dose escalation of KTX-2001 monotherapy (Part A) and in combination with darolutamide, an oral, nonsteroidal androgen receptor inhibitor (Part B).

"KTX-2001 is a first-in-class NSD2 inhibitor targeting a long-recognized epigenetic driver of prostate cancer biology," said Terry Connolly, Ph.D., President and Chief Executive Officer, K36 Therapeutics. "Despite recent therapeutic advances, many patients ultimately exhaust effective options. This trial introduces a novel epigenetic mechanism with the potential to open an entirely new treatment paradigm for men with advanced disease."

In parallel with advancing its lead clinical programs, K36 recently appointed Shinta Cheng, M.D., Ph.D., as Chief Medical Officer. Dr. Cheng brings more than 20 years of global oncology and hematology drug development experience, including leadership roles at SpringWorks Therapeutics, Johnson & Johnson, and Bristol Myers Squibb, with deep expertise in prostate cancer, including leading the development of apalutamide and niraparib.

"The advancement of KTX-2001 highlights both the urgent need for new therapies in advanced prostate cancer and the promise of a first-in-class NSD2-targeted approach. I am excited to have joined K36 Therapeutics at this pivotal moment as we advance KTX-2001, our second NSD2 inhibitor with potential across a broader range of solid tumors," said Dr. Cheng.

"Site activation is progressing ahead of schedule, with more than 75% of sites targeting activation by the end of the month and enrollment into subsequent cohorts underway. This early momentum reflects strong clinical interest in oral epigenetic modifier therapies for metastatic castration-resistant prostate cancer and underscores the urgent need for new treatment options for patients," said Jason Redman, M.D., Prostate Program Medical Director at K36 Therapeutics.

About the KTX-2001 Phase 1 Clinical Trial (STRIKE-001)
STRIKE-001 (NCT07103018) is a multi-center, open-label dose escalation evaluating KTX-2001 as a monotherapy (Part A) and in combination with darolutamide (Part B).

Part A is designed to evaluate the safety, tolerability, maximum tolerated dose, and recommended Phase 2 dose(s) of KTX-2001 monotherapy. Part B will evaluate the safety and tolerability of KTX-2001 plus darolutamide to determine the recommended Phase 2 dose(s) for the combination. Secondary objectives include assessments of pharmacokinetics, pharmacodynamics, and preliminary clinical activity. K36 expects to enroll approximately 140 patients with mCRPC who have received prior androgen receptor inhibitors and prior chemotherapy.

KTX-2001 is a small molecule, selective inhibitor of nuclear receptor binding SET domain protein 2 (NSD2, also known as multiple myeloma [MM] SET domain-containing protein [MMSET]/Wolf-Hirschhorn syndrome candidate 1 protein [WHSC1]). KTX-2001 inhibits NSD2-mediated methylation of histone H3 at lysine 36 (H3K36), disrupting aberrant NSD2-dependent oncogenic pathways.

(Press release, K36 Therapeutics, FEB 5, 2026, https://www.prnewswire.com/news-releases/k36-therapeutics-completes-dosing-of-first-cohort-in-phase-1-clinical-trial-of-ktx-2001-in-prostate-cancer-announces-new-cmo-302680429.html [SID1234662517])