On January 12, 2026 Autolus Therapeutics plc (Nasdaq: AUTL), a commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, reported preliminary unaudited net product revenue from sales of AUCATZYL (obecabtagene autoleucel; obe-cel) for the fourth quarter and full year of 2025. The Company also announced pipeline advancement updates and anticipated future milestones.

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AUCATZYL 2025 Preliminary Unaudited Net Product Revenues, Commercial Updates and 2026 Revenue Guidance

Dr. Christian Itin, Chief Executive Officer of Autolus, said: "We had a successful launch of AUCATZYL in the US with full year sales well above expectations and more than 60 centers offering treatment. We established reliable, high-quality product delivery with short and consistent turn-around time. Parallel to the launch and independent from the Company, the ROCCA consortium collected real-world data for AUCATZYL in adult r/r B-ALL patients and presented initial data at ASH (Free ASH Whitepaper) 2025. The real-world data confirmed a high level of clinical activity and a favorable safety profile for AUCATZYL consistent with prior clinical trial results, and we believe this positive customer experience will be a key driver for the future growth of AUCATZYL in 2026.

"In addition to our launch of AUCATZYL in the US, we obtained regulatory approvals in the UK and EU. We successfully navigated the challenging pricing and reimbursement process with NICE, establishing cost effectiveness of AUCATZYL for the NHS and initiating our commercial launch in the UK in December under routine commissioning – a first for a CAR T therapy in the UK."

Based on preliminary unaudited financial information, Autolus expects net product revenue from sales of AUCATZYL to be approximately $24 million1 for the fourth quarter of 2025. Net product revenues for the full year of 2025, the first year of commercial sales, are expected to be approximately $75 million1. The Company plans to report its fourth quarter and full year 2025 financial results in March 2026.

For 2026, the Company projects net product revenue for AUCATZYL of between $120 million to $135 million.

Pipeline Updates and Upcoming Clinical Milestones

Dr. Matthias Will, Chief Development Officer of Autolus, said: "In 2025 we reported strong initial clinical data for the Phase 1 CATULUS trial in pediatric r/r B-ALL patients and in the Phase 1 CARLYSLE trial in patients with severe lupus erythematosus. Both data sets form a compelling basis for progressing those studies in pediatric B-ALL and in lupus nephritis to Phase 2. With this strong foundation of clinical data, Phase 2 studies enrolling and demonstrated commercial and manufacturing capabilities, we believe Autolus is well positioned to drive growth with obe-cel in additional and significant indications."

Obe-cel data in pediatric r/r B-ALL

Preliminary data from the CATULUS Phase 1 trial of obe-cel in pediatric relapsed or refractory (r/r) B-ALL patients were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Obe-cel demonstrated high remission rates in pediatric patients with high-risk r/r B-ALL with overall response rate (ORR) of 95.5%. Low rates of high-grade cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were observed, consistent with obe-cel’s adult safety profile. Autolus is now advancing obe-cel into the Phase 2 portion of the trial and expects to have the trial fully enrolled in the first half of 2027.
Obe-cel in lupus nephritis (LN)

Data from the ongoing Phase 1 CARLYSLE trial in patients with severe refractory systemic lupus erythematosus (srSLE) were reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. All patients show deep B-cell depletion after infusion, suggesting an immune reset. No ICANS or high-grade CRS were observed in the nine patients evaluable for safety.
Data support progressing obe-cel as a treatment for LN and 50 million cells was selected as the recommended Phase 2 dose. Autolus has previously aligned with the US Food and Drug Administration (FDA) on a Phase 2 trial design in LN and potential registrational path to approval. The LUMINA trial is now enrolling.
Obe-cel in progressive multiple sclerosis (MS)

Autolus is advancing obe-cel into initial clinical development to explore treatment in progressive MS. The first patient in the BOBCAT trial was dosed in October 2025. The Phase 1 trial, expected to include up to 18 adult patients, will determine the safety, tolerability, and preliminary efficacy of obe-cel in participants with refractory progressive forms of MS. Initial data from the trial are expected to be reported at the end of 2026.
AUTO8 in light-chain amyloidosis

The first patient was dosed in the Phase 1 ALARIC trial in AL amyloidosis and initial data is expected to be reported at the end of 2026.
Operational Updates & 2026 Outlook

Dr. Christian Itin concluded: "Our 2026 commercial focus for AUCATZYL is to build on the strong center presence and positive physician experience to drive top line growth, while improving margins by reducing our manufacturing costs per batch. Our development focus is on progressing our pivotal pediatric ALL CATLULUS and lupus nephritis LUMINA studies and the exploratory BOBCAT study in progressive multiple sclerosis. Our plans for innovation will focus on manufacturing technology and European market access."

Increasing patient numbers in 2026 will improve manufacturing plant utilization and together with operational efficiencies, Autolus expects a shift from previously reported negative gross margin to positive gross margin in 2026.

Autolus has initiated an overall manufacturing life cycle plan to facilitate additional cost reductions and gross margin improvements as the Company plans to expand obe-cel into new indications and pursue larger market opportunities. The initiatives are focused on: 1) optimizing the Company’s current manufacturing operating model; 2) enhancing automation opportunities for the Company’s existing manufacturing process; and 3) developing a next-generation manufacturing platform with a step change in the cost and capacity profile. The Company plans to provide a detailed update on these plans in mid-2026.

Based on current operating plans, including anticipated AUCATZYL net revenues, Autolus expects that its current and projected cash, cash equivalents and marketable securities will be sufficient to fund the Company’s operations into Q4 2027.

(Press release, Autolus, JAN 12, 2026, View Source [SID1234661938])

On January 12, 2026 Aim Immunotech presented its corporate presentation.

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(Presentation, AIM ImmunoTech, JAN 12, 2026, View Source [SID1234661937])

On January 12, 2026 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, reported its 2026 strategic priorities and key milestones anticipated during the year. Members of the company’s management team will present this update at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026, at 8:15 a.m. PT / 11:15 a.m. ET.

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"In 2025, Agios delivered another year of strong and consistent execution across our portfolio, marking meaningful progress toward our goal of becoming a sustainable and diversified rare disease company," said Brian Goff, Chief Executive Officer, Agios. "Last year culminated in the historic U.S. approval of AQVESME (mitapivat), our pyruvate kinase (PK) activator and the only medicine approved to treat anemia in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. This approval brings a new, disease-modifying oral option to people living with this debilitating and deadly rare blood disorder.

"Entering 2026, the company is at an important inflection point. We will deliver a high-impact U.S. launch of AQVESME in thalassemia, seek to expand our PK activation franchise into additional high-value indications such as sickle cell disease and lower-risk myelodysplastic syndromes, and advance our promising early-stage pipeline with the potential to further diversify across hematologic and rare diseases. We also remain focused on disciplined capital allocation and operational efficiency to support our long-term sustainability. With strong momentum and a clear roadmap, Agios enters the year positioned to deliver transformative innovation and meaningful impact for patients living with rare diseases," Mr. Goff added.

Anticipated 2026 Milestones
Thalassemia

In December 2025, the U.S. Food and Drug Administration (FDA) approved AQVESME for the treatment of anemia in adults with alpha- or beta-thalassemia. AQVESME is the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.
Agios expects AQVESME to become available in the U.S. in late January 2026 following implementation of the AQVESME Risk Evaluation and Mitigation Strategy (REMS) program. Commercial launch activities are already underway and will continue throughout the year.
Sickle Cell Disease

Topline results from the RISE UP Phase 3 trial of mitapivat in sickle cell disease were reported in November 2025. Agios anticipates having a pre-supplemental New Drug Application (sNDA) meeting with the FDA in the first quarter of 2026, and plans to submit a U.S. marketing application for mitapivat in sickle cell disease following that engagement.
Enrollment in the Phase 2 sickle cell disease trial of tebapivat, Agios’ more potent, once-daily oral PK activator, was initiated in 2025. Agios expects to report topline results from this trial in the second half of 2026.
Lower-Risk Myelodysplastic Syndromes (LR-MDS)

Enrollment in the Phase 2b LR-MDS trial of tebapivat was completed in 2025. Agios expects to report topline results from this trial in the first half of 2026.
Polycythemia Vera (PV)

Agios expects to report topline results from a Phase 1 healthy volunteer trial of AG-236, a small interfering RNA (siRNA) targeting TMPRSS6 as a potential treatment for PV, in the first half of 2026.
Phenylketonuria (PKU)

With dosing completed in the Phase 1 single- and multiple-ascending-dose trial of AG-181, a phenylalanine hydroxylase (PAH) stabilizer, in healthy volunteers, Agios expects to initiate a Phase 1b proof-of-mechanism trial of AG-181 in patients with PKU in the first half of 2026 and to confirm proof of mechanism in the second half of 2026.
Presentation at 44th Annual J.P. Morgan Healthcare Conference
Members of Agios’ management team will present at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026, at 8:15 a.m. PT / 11:15 a.m. ET. The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be archived on the company’s website for at least two weeks following the presentation.

About AQVESME (mitapivat)
U.S. INDICATION
AQVESME is indicated for the treatment of anemia in adults with alpha- or beta-thalassemia.

U.S. IMPORTANT SAFETY INFORMATION
BOXED WARNING: HEPATOCELLULAR INJURY

AQVESME can cause serious hepatocellular injury. Measure liver laboratory tests (ALT, AST, alkaline phosphatase and total bilirubin with fractionation) at baseline and every 4 weeks for 24 weeks and then as clinically indicated. Avoid use of AQVESME in patients with cirrhosis. Discontinue AQVESME if hepatic injury is suspected.
Because of the risk of hepatocellular injury, AQVESME is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the AQVESME REMS.

WARNINGS AND PRECAUTIONS

Hepatocellular Injury
AQVESME can cause hepatocellular injury. Avoid use of AQVESME in patients with cirrhosis. In patients with thalassemia treated with AQVESME, liver injury with and without jaundice has been observed within the first 6 months of exposure. Obtain liver tests (including ALT, AST, alkaline phosphatase, total bilirubin with fractionation) prior to the initiation of AQVESME, then every 4 weeks for the first 24 weeks, and as clinically indicated thereafter. Interrupt AQVESME if clinically significant increases in liver tests are observed or alanine aminotransferase is >5 times the upper limit of normal (ULN). Complete a comprehensive evaluation to rule out other causes of liver injury when drug-induced liver injury is suspected. Discontinue AQVESME if hepatocellular injury due to AQVESME is suspected.

Symptoms and signs of early liver injury may mimic those of thalassemia. Advise patients to report new or worsening symptoms of loss of appetite, nausea, right-upper-quadrant abdominal pain, vomiting, scleral icterus, jaundice, or dark urine while on AQVESME treatment.

During the double-blind period, 2 of 301 patients (0.66%) with thalassemia treated with AQVESME experienced adverse reactions suggestive of hepatocellular injury. Three additional patients experienced adverse reactions suggestive of hepatocellular injury during the open-label extension periods after switching from placebo to AQVESME. Of these 5 patients, 2 had serious liver injury requiring hospitalization, including 1 patient who developed jaundice (peak bilirubin 32 mg/dL). Another patient developed jaundice (peak bilirubin 4 mg/dL) without requiring hospitalization. These reactions were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5×ULN with or without jaundice. All patients discontinued treatment with AQVESME, and these reactions improved upon treatment discontinuation.

AQVESME REMS
AQVESME is available only through a restricted program under a REMS called the AQVESME REMS because of the risk of hepatocellular injury.

ADVERSE REACTIONS
The most common adverse reactions among patients taking AQVESME were headache and insomnia.

DRUG INTERACTIONS

Strong CYP3A Inhibitors and Inducers: Avoid concomitant use.
Moderate CYP3A Inhibitors: Avoid concomitant use.
Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, see full Prescribing Information for recommended dosage for drug interactions with moderate CYP3A inducers.
Sensitive CYP3A Substrates, including hormonal contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index.
CYP2B6, CYP2C, and UGT1A1 Substrates: Monitor patients for efficacy of the substrates with narrow therapeutic index.
P-gp Substrates: Monitor patients for adverse reactions of the substrates with narrow therapeutic index.
HEPATIC IMPAIRMENT
Avoid use of AQVESME in patients with cirrhosis (Child-Pugh Class A, B, or C).

Please see full Prescribing Information for AQVESME, including Boxed Warning.

(Press release, Agios Pharmaceuticals, JAN 12, 2026, View Source [SID1234661936])

On January 12, 2026 ADC therapeutics presented its corporate presentation.

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(Presentation, ADC Therapeutics, JAN 12, 2026, View Source [SID1234661935])

On January 12, 2026 Actuate Therapeutics, Inc. (NASDAQ: ACTU) ("Actuate" or the "Company"), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), reported that new additional and promising patient survival and biomarker data from treatment with elraglusib for metastatic pancreatic cancer were featured in oral and poster presentations at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI) on January 9, 2026. The presentations highlighted new, expanded data from the Company’s Phase 2 clinical program (Actuate 1801-Part 3B) evaluating elraglusib in combination with gemcitabine/nab-paclitaxel (GnP), a first-line chemotherapy regimen for metastatic pancreatic cancer, providing additional evidence that suggests that elraglusib combined with GnP is associated with improved survival when compared with GnP alone.

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"Pancreatic cancer continues to have a high unmet medical need, particularly at the metastatic stage," said Dr. Devalingam Mahalingam, MD, PhD. "The results from this Phase 2 study suggest that adding elraglusib to standard gemcitabine and nab-paclitaxel chemotherapy may improve survival outcomes while maintaining a manageable safety profile. The scope of this trial, which enrolled 286 patients across 60 sites in six countries, underscores the robustness of the dataset. We are grateful to the patients and their families whose participation made this research possible."

"Metastatic pancreatic cancer remains one of the most difficult cancers to treat, with few effective first-line options," said Dan Schmitt, Chief Executive Officer of Actuate Therapeutics. "In this randomized Phase 2 study, the addition of elraglusib to gemcitabine and nab-paclitaxel resulted in a meaningful improvement in overall survival compared with chemotherapy alone. As of November 2025, seventeen patients are still alive in the elraglusib/GnP arm, and three of them have remarkably passed the 24-month mark on first-line treatment, while no patients remain on GnP treatment alone. Current patient survival for metastatic pancreatic cancer is less than 12 months, so our findings are especially encouraging. We look forward to building from this data and continuing to advance elraglusib for patients with this devastating disease."

Key Findings:

Efficacy: The primary efficacy endpoints were met in this study:

· The median overall survival (OS) showed significant benefit in the elraglusib/GnP vs GnP alone arms (mOS 10.1 months vs. 7.2 months, p=0.02, HR=0.62)
· 12-month survival rate doubled to 44.4% (elraglusib/GnP) vs 22.3% (GnP)
· 24-month survival rate increased fivefold to 12.9% in the elraglusib/GnP arm compared to 2.6% in the GnP arm, emphasizing the potential for long-term clinical benefit.

Safety: The safety and tolerability profile of elraglusib was consistent with previously reported data at the 9.3 mg/kg dose, and no new safety signals were identified:

· Serious TEAE were similar between the elraglusib/GnP (56.1%) and the GnP alone arm (56.4%),
· TEAE resulting in death were similar between treatment arms: elraglusib/GnP (12.3%) and GnP alone (16.7%)
· Grade-3 or higher TEAE leading to stoppage of any study drug: elraglusib/GnP (16.8%) and GnP alone (21.8%)

Genomic biomarkers: Several mutations commonly associated with pancreatic cancer were identified as potential predictive biomarkers of overall survival in the elraglusib/GnP arm, but not in the GnP-alone arm. Actionable mutations like KRAS or ARID1A could suggest novel drug combinations for future clinical studies.

Immunological biomarkers: The percentage of CD8+ and Granzyme B+ cells, as well as NK cells, was increased in tumors obtained from elraglusib/GnP-treated patients but not in GnP-treated patients, suggesting that combination therapy may potentiate improved anti-tumor immune response.

Oral Presentation

Title: Results from the randomized Phase 2 study (1801 Part 3B) of elraglusib plus gemcitabine/nab-paclitaxel (GnP) versus GnP in previously untreated metastatic pancreatic ductal adenocarcinoma (mPDAC).
Abstract: 653
Session: Rapid Oral Abstract Session B: Cancers of the Pancreas, Small Bowel, and Hepatobiliary Tract
Date/Time: January 9, 2026, 4:15 PM-5:00 PM (PST)
First Author: Devalingam Mahalingam, MD, PhD, Gastrointestinal Oncologist and Professor of Medicine at Northwestern University Feinberg School of Medicine

Poster Presentation

Title: Mutational analysis and identification of potential biomarkers in patients with metastatic pancreatic cancer treated with the combination of the GSK-3 inhibitor elraglusib and gemcitabine/nab-paclitaxel in the 1801 Part 3β Phase 2 study.
Abstract: 761
Poster Bd: L5
Session: Poster Session B: Cancers of the Pancreas, Small Bowel, and Hepatobiliary Tract
Date/Time: January 9, 2026, 11:30 AM-1:00 PM; 5:00 PM-6:00 PM (PST)
First Author: Andrey Ugolkov, MD, PhD, Senior Director, Clinical Science at Actuate Therapeutics

About Actuate-1801 Part 3B Study

The Actuate-1801 Part 3B study (NCT03678883) is a randomized, controlled Phase 2 trial of elraglusib with GnP versus GnP alone in first-line mPDAC. The trial enrolled 286 mPDAC patients with no prior systemic treatment for metastatic disease, who were randomized 2:1 to the elraglusib treatment arm (elraglusib/GnP) or the control arm (GnP alone). Elraglusib is administered at a dose of 9.3 mg/kg by IV infusion on Day 1 of each week of a 28-day cycle. The primary endpoint for this study is median overall survival, with OS summarized throughout the study by estimates of 1-year survival. Secondary endpoints are DCR, ORR, PFS, and AE.

(Press release, Actuate Therapeutics, JAN 12, 2026, View Source [SID1234661934])