On November 3, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported that data from the ongoing Phase 1b study of R2891, a potent and selective inhibitor of dual interleukin receptor-associated kinases 1 and 4 (IRAK1/4), in patients with lower-risk myelodysplastic syndrome (MDS) who are relapsed or refractory (R/R) to prior therapies will be presented in an oral session at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition on Sunday, December 7, 2025. In addition, the ASH (Free ASH Whitepaper) Annual Meeting will feature four poster presentations with data for REZLIDHIA (olutasidenib) for the treatment of R/R mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML). The ASH (Free ASH Whitepaper) Annual Meeting is being held December 6-9, 2025, in Orlando, Florida and virtually.

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 very pleased to have the opportunity to highlight our hematology and oncology portfolio at ASH (Free ASH Whitepaper) this year," said Lisa Rojkjaer, M.D, Rigel’s chief medical officer. "In particular, we are delighted that updated results from our Phase 1b study in patients with relapsed or refractory lower-risk MDS have been accepted for oral presentation. Despite the availability of approved agents, there remains an unmet need for additional therapies to treat patients with transfusion dependent lower-risk MDS, and we look forward to advancing the dose expansion phase of the study to completion."

ASH Annual Meeting abstracts may be accessed online at www.hematology.org. Details of the oral and poster presentations, which will be available in the poster hall and via the virtual event platform, are as follows:

Oral Presentation

Sunday, December 7, 2025, 9:45am to 10:00am ET
Publication #: 489
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML
Presentation Title: An Update of Safety and Efficacy Results from a Phase 1b Study of R289, a Dual IRAK 1/4 Inhibitor, in Patients with Relapsed/Refractory (R/R) Lower Risk Myelodysplastic Syndrome (LR-MDS)
Presenter: Guillermo Garcia-Manero, M.D.

As of the data cutoff date (July 15, 2025), 33 patients were enrolled in the dose escalation part of the study. Patients had a median age of 75 with a median of 3 prior therapies and 61% were high transfusion burden at baseline.
Patients received R289 at doses ranging from 250 mg QD (once daily) to 500 mg BID (twice daily). For the 500 mg BID dose group, five patients were not yet evaluable (<16 weeks follow up) for determination of hematologic responses and one patient withdrew consent.
R289 was generally well tolerated across all dose groups, with the most frequent treatment emergent adverse events (≥20%) being diarrhea (28.1%), constipation/fatigue (25% each), and creatinine/alanine aminotransferase (ALT) increased (21.9% each), the majority being Grade 1/2. One (1) dose limiting toxicity (DLT) (Grade 4 aspartate aminotransferase (AST) increase/Grade 3 ALT increase) was reported in the 750 mg dose group.
For evaluable transfusion dependent patients (≥16 weeks follow up) at dose levels of at least 500 mg QD and higher, 4/13 patients (31%) achieved durable red blood cell transfusion independence (RBC-TI) for >8 weeks (500 mg QD [1/3], 750 mg QD [2/5], 500/250 mg QD [1/5]). Duration of RBC-TI was >16 weeks in 3 patients, >24 weeks in 2 patients, and >12 months in 1 patient. The median time to onset of RBC-TI was 2.2 months and the median duration of RBC-TI was 24.3 weeks.
All responding patients had R835 plasma concentrations similar to those at which ≥50% LPS-induced inhibition of cytokine release was observed in healthy volunteers, indicating a potential threshold for dose response (≥500 mg QD).
Updated data as of an October 28, 2025, data cutoff will be presented during the oral presentation.
Poster Presentations

Saturday, December 6, 2025, 5:30pm to 7:30pm ET
Publication #: 1659
Title: Clinical Characteristics and Response in Olutasidenib-Treated Relapsed/Refractory mIDH1 Acute Myeloid Leukemia (AML) Patients With Stable Disease Following Two Treatment Cycles
Presenter: Justin M. Watts, M.D.

In the pivotal cohort of the Phase 2 registrational study, of 147 patients with R/R mIDH1 AML who received olutasidenib, 36 (24%) patients maintained stable disease (SD) after 2 cycles of treatment. Treatment duration ranged from 2.6 to 51.1 months. In these 36 patients, the subsequent response rate was 33% (n=12), including 6 (17%) complete remission (CR), 2 (6%) CR with partial hematologic recovery (CRh), 1 (3%) CR with incomplete recovery, 1 (3%) morphologic leukemia-free state, and 2 (6%) partial remission.
Median time to best response from the start of treatment was 3.7 months (range: 2.8-5.7). 8 patients (22%) remained in SD and 16 (44%) had subsequent disease progression on study.
In the 12 late responders, median duration of response (DOR), duration of CR/CRh, and duration of CR were 9.9 months, 17.3 months, and not reached, respectively. Additionally, of the late responders who were transfusion dependent for platelets (n=6) or red blood cells (n=9) at baseline, 5 (83%) and 8 (89%), respectively, became transfusion independent.
Patients who achieved any late response had a longer median treatment duration (10.1 months) than non-responders. Median overall survival (OS) for late responders was 23.9 months and 32.7 months for those with CR/CRh.
Patients with SD after 2 cycles of olutasidenib may experience meaningful clinical benefit with continued treatment, as one-third of these patients subsequently achieved a late response, resulting in a lower risk of death compared to patients with no later response. These findings suggest that early SD may not predict treatment failure and support continuing olutasidenib for at least 6 cycles or until disease progression.
Sunday, December 7, 2025, 6:00pm to 8:00pm ET
Publication #: 4616
Title: Assessment of Real-World Treatment Patterns and Outcomes of Olutasidenib in Patients with Mutated Isocitrate Dehydrogenase 1 Acute Myeloid Leukemia Previously Treated with Venetoclax Using Electronic Health Record Data
Presenter: Catherine Lai, M.D., MPH

This retrospective cohort study analyzed data from Loopback Analytics’ electronic health records data in the U.S. until September 2024, incorporating structured clinical data and abstracted data from physician notes. Fourteen olutasidenib-treated patients in the Loopback database met inclusion criteria for the study.
The overall response rate (ORR) was 50% (7/14) and the composite complete remission (CRc) rate was 36% (5/14). Among patients who achieved any response, 86% (6/7) received venetoclax immediately prior to olutasidenib.
Median OS from olutasidenib initiation in the full cohort was 12.2 months, with the proportion of patients surviving 6, 9, and 12 months estimated to be 88%, 70%, and 53%, respectively.
In this real-world cohort, despite the small sample size, 50% of patients responded to post-venetoclax olutasidenib, consistent with the clinical efficacy observed in the pivotal Phase 2 trial. These findings support the use of olutasidenib as a viable therapeutic option in post-venetoclax treatment settings.
Publication #: 3439
Title: Analysis of Hematologic Improvement (HI) by Time to Response in Relapsed/Refractory Acute Myeloid Leukemia (AML) Patients Treated with Olutasidenib
Presenter: Shira N. Dinner, M.D

In the pivotal cohort of the Phase 2 registrational study (n=147), increases in hemoglobin were seen early in the course of treatment and levels continued to increase over 12 cycles. Similarly, platelet counts increased and blast percentages decreased over the course of treatment.
A total of 71 patients (48%) achieved an overall response; 47 patients (32%) achieved CR and 51 (35%) achieved CR/CRh. Among CR/CRh responders, 28 (55%) achieved a response in <2 months, 17 (33%) from 2 to 4 months, and 6 (12%) at >4 months.
Patients with a longer time to response tended to have lower baseline platelet counts and higher bone marrow blast percentages compared with earlier responders, suggesting lower hematopoietic reserve and greater disease burden.
In 37 patients (25%) who had a best response of SD, several showed improvement in platelets and hemoglobin levels by end of treatment. 7 of 21 (33%) patients with prior platelet transfusion dependence became independent and 7 of 23 (30%) with prior red blood cell transfusion dependence became independent.
This report highlights the hematological responses to olutasidenib in these patients with R/R mIDH1 AML and suggests that continuing olutasidenib treatment beyond 2 cycles may offer hematologic benefits, even in the absence of an early clinical response.
Monday, December 8, 2025, 6:00pm ET to 8:00pm ET
Publication #: 5213
Title: Olutasidenib Monotherapy in Patients With mIDH1 Acute Myeloid Leukemia Who Received Prior Intensive Chemotherapy
Presenter: Jay Yang, M.D.

In the pivotal cohort of the Phase 2 registrational study (n=147), in patients with prior intensive chemotherapy (IC), the ORR was 50% with 35/105 (33%) achieving CR and 38/105 (36%) achieving CR/CRh. Median DOR was 15.5 months, with median duration of CR not reached and CR/CRh of 17.6 months.
After a median follow up of 37.3 months, the median OS was 12.5 months. In comparison, in the 38 patients who received prior non-intensive therapy the ORR was 42%; 9/38 achieved CR and 10/38 achieved CR/CRh. The median DOR in this group was 16.2 months, with a median duration of CR and CR/CRh of 28.1 months and 29.0 months, respectively.
Among this cohort of patients with R/R mIDH1 AML who had received prior IC, treatment with olutasidenib monotherapy produced clinically meaningful response rates that closely align with those observed in the overall population, with durable responses and acceptable tolerability.
About R289
R289 is a prodrug of R835, an IRAK1/4 dual inhibitor, which has been shown in preclinical studies to block inflammatory cytokine production in response to toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to various inflammatory conditions. Chronic stimulation of both these receptor systems is thought to cause the pro-inflammatory environment in the bone marrow responsible for persistent cytopenias in lower-risk MDS patients.2

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 22,010 new cases in the United States, most in adults, in 2025.3

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.4,5 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.6 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 REZLIDHIA

INDICATION
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.

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, can occur with REZLIDHIA treatment. Symptoms may include dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney injury, hypotension, fever, and weight gain. If differentiation syndrome is suspected, withhold REZLIDHIA and initiate treatment with corticosteroids and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome
REZLIDHIA can cause differentiation syndrome. In the clinical trial of REZLIDHIA in patients with relapsed or refractory AML, differentiation syndrome occurred in 16% of patients, with grade 3 or 4 differentiation syndrome occurring in 8% of patients treated, and fatalities in 1% of patients. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal. Symptoms of differentiation syndrome in patients treated with REZLIDHIA included leukocytosis, dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney injury, fever, edema, pyrexia, and weight gain. Of the 25 patients who experienced differentiation syndrome, 19 (76%) recovered after treatment or after dose interruption of REZLIDHIA. Differentiation syndrome occurred as early as 1 day and up to 18 months after REZLIDHIA initiation and has been observed with or without concomitant leukocytosis.

If differentiation syndrome is suspected, temporarily withhold REZLIDHIA and initiate systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours) for a minimum of 3 days and until resolution of signs and symptoms. If concomitant leukocytosis is observed, initiate treatment with hydroxyurea, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms. Differentiation syndrome may recur with premature discontinuation of corticosteroids and/or hydroxyurea treatment. Institute supportive measures and hemodynamic monitoring until improvement; withhold dose of REZLIDHIA and consider dose reduction based on recurrence.

Hepatotoxicity
REZLIDHIA can cause hepatotoxicity, presenting as increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), increased blood alkaline phosphatase, and/or elevated bilirubin. Of 153 patients with relapsed or refractory AML who received REZLIDHIA, hepatotoxicity occurred in 23% of patients; 13% experienced grade 3 or 4 hepatotoxicity. One patient treated with REZLIDHIA in combination with azacitidine in the clinical trial, a combination for which REZLIDHIA is not indicated, died from complications of drug-induced liver injury. The median time to onset of hepatotoxicity in patients with relapsed or refractory AML treated with REZLIDHIA was 1.2 months (range: 1 day to 17.5 months) after REZLIDHIA initiation, and the median time to resolution was 12 days (range: 1 day to 17 months). The most common hepatotoxicities were elevations of ALT, AST, blood alkaline phosphatase, and blood bilirubin.

Monitor patients frequently for clinical symptoms of hepatic dysfunction such as fatigue, anorexia, right upper abdominal discomfort, dark urine, or jaundice. Obtain baseline liver function tests prior to initiation of REZLIDHIA, at least once weekly for the first two months, once every other week for the third month, once in the fourth month, and once every other month for the duration of therapy. If hepatic dysfunction occurs, withhold, reduce, or permanently discontinue REZLIDHIA based on recurrence/severity.

ADVERSE REACTIONS
The most common (≥20%) adverse reactions, including laboratory abnormalities, were aspartate aminotransferase increased, alanine aminotransferase increased, potassium decreased, sodium decreased, alkaline phosphatase increased, nausea, creatinine increased, fatigue/malaise, arthralgia, constipation, lymphocytes increased, bilirubin increased, leukocytosis, uric acid increased, dyspnea, pyrexia, rash, lipase increased, mucositis, diarrhea and transaminitis.

DRUG INTERACTIONS

Avoid concomitant use of REZLIDHIA with strong or moderate CYP3A inducers.
Avoid concomitant use of REZLIDHIA with sensitive CYP3A substrates unless otherwise instructed in the substrates prescribing information. If concomitant use is unavoidable, monitor patients for loss of therapeutic effect of these drugs.
LACTATION
Advise women not to breastfeed during treatment with REZLIDHIA and for 2 weeks after the last dose.

GERIATRIC USE
No overall differences in effectiveness were observed between patients 65 years and older and younger patients. Compared to patients younger than 65 years of age, an increase in incidence of hepatotoxicity and hypertension was observed in patients ≥65 years of age.

HEPATIC IMPAIRMENT
In patients with mild or moderate hepatic impairment, closely monitor for increased probability of differentiation syndrome.

Click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

REZLIDHIA is a registered trademark of Rigel Pharmaceuticals, Inc.

(Press release, Rigel, NOV 3, 2025, View Source [SID1234659279])

On November 3, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported it will webcast management participation as follows:

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!

Jefferies London Healthcare Conference at 3:30 p.m. GMT (10:30 a.m. ET) on Monday, November 17, 2025
7th Annual Wolfe Research Healthcare Conference at 9:20 a.m. ET on Monday, November 17, 2025
8th Annual Evercore Healthcare Conference at 1:20 p.m. ET on Tuesday, December 2, 2025
Citi 2025 Global Healthcare Conference at 10:30 a.m. ET on Wednesday, December 3, 2025

The sessions may be accessed from the "Investors & Media" page of Regeneron’s website at View Source Replays and transcripts of the webcasts will be archived on the Company’s website for at least 30 days.

(Press release, Regeneron, NOV 3, 2025, View Source [SID1234659278])

On November 3, 2025 Pyxis Oncology, Inc. (Nasdaq: PYXS), a clinical-stage company developing next-generation therapeutics for difficult-to-treat cancers, reported a business update, and announced financial results for the quarter ended September 30, 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!

"In the dynamic landscape of emerging clinical-stage therapies for patients with recurrent and metastatic head and neck squamous cell carcinoma, a significant unmet medical need remains despite the potential improvements in treatment options," said Lara S. Sullivan, M.D., President, Chief Executive Officer and Chief Medical Officer of Pyxis Oncology. "We look forward to presenting our preliminary data from the ongoing clinical studies evaluating MICVO as a novel potential treatment option for recurrent and metastatic head and neck squamous cell carcinoma. We believe that the breadth of the MICVO clinical program, encompassing monotherapy and combination approaches, holds significant promise and this inflection point will further underscore our first-in-concept ADC’s potential to improve outcomes across multiple lines of therapy."

Pipeline & Corporate Updates
•
Pyxis Oncology expects to report preliminary data from the ongoing Phase 1 clinical studies of micvotabart pelidotin (MICVO) in patients with recurrent and metastatic head and neck squamous cell carcinoma (R/M HNSCC) in 4Q25.

o
Clinical update to focus on preliminary data from the Phase 1 monotherapy dose expansion study of MICVO for 2L/3L R/M HNSCC patients, including both the post platinum and anti-PD(L)-1 experienced arm and the post EGFRi and anti-PD(L)-1 experienced arm.
o
Additional preliminary clinical data from the Phase 1/2 combination dose escalation study of MICVO and KEYTRUDA (pembrolizumab) for 1L/2L+ R/M HNSCC patients will also be provided. The combination study is part of a Clinical Trial Collaboration Agreement with Merck (known as MSD outside of the US and Canada).
o
Pyxis Oncology expects to announce next steps in the clinical development plan for MICVO for R/M HNSCC along with the preliminary data update.

•
Pyxis Oncology presented new translational data in October 2025 in two posters at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025 and in six posters at the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference, as well as three clinical trial in progress posters at ESMO (Free ESMO Whitepaper). The presentation posters at ESMO (Free ESMO Whitepaper) and AACR (Free AACR Whitepaper)-NCI-EORTC provided deeper insights into the pharmacodynamic responses of tumors to MICVO as well as MICVO’s unique mechanism of action and its potential to exert anti-tumor activity through three mechanisms: direct tumor cell killing, bystander killing and immunogenic cell death.
o
Translational findings highlighted MICVO’s effects on tumor microenvironment remodeling and immune activation, further reinforcing the potential benefit of MICVO as both a monotherapy and in combination with anti-PD1 therapy.
o
Observations included changes in circulating tumor DNA (ctDNA) tumor fraction (TF) to the vast majority of 37 clinical samples tested. Notably, reduction in ctDNA TF after treatment with MICVO, particularly in HNSCC and at the 5.4 mg/kg dose, support a positive molecular response to MICVO and strengthen rationale for continued development for this tumor type and dose in the monotherapy dose expansion study.
o
Additionally, features observed in nonclinical samples of the stromal architecture detected using AI-enabled hyper-resolution digital pathology may correlate with sensitivity to MICVO – a finding that may be unique compared to tumor cell surface targeting ADCs, due to MICVO’s targeting of a non-cellular structural component of the extracellular matrix.

Third Quarter 2025 Financial Results

•
As of September 30, 2025, Pyxis Oncology had cash and cash equivalents, including restricted cash, and short-term investments, of $77.7 million. The Company believes that its current cash, cash equivalents, and short-term investments will be sufficient to fund its operations into the second half of 2026.

•
Research and development expenses were $17.8 million for the quarter ended September 30, 2025, compared to $17.7 million for the quarter ended September 30, 2024. MICVO program-specific research and development costs increased by $2.0 million, primarily due to a $1.0 million increase in contract manufacturing costs and a $1.3 million increase in clinical trial related expenses related to monotherapy and combination therapy of MICVO. The increase in expenses was partially offset by a $1.8 million reduction in expenses related to PYX-106, as the clinical development of PYX-106 was paused in December 2024.

•
General and administrative expenses were $5.6 million for the quarter ended September 30, 2025, compared to $6.0 million for the quarter ended September 30, 2024. The decrease was primarily due to lower corporate insurance costs and a decrease in legal, professional and consulting fees.

•
Net loss was $22.0 million, or ($0.35) per common share, for the quarter ended September 30, 2025, compared to $21.2 million, or ($0.35) per common share, for the quarter ended September 30, 2024. Excluding non-cash stock-based compensation expense, the net loss for the quarter ended September 30, 2025 was $18.9 million, compared to a net loss of $18.2 million for the quarter ended September 30, 2024.

•
As of October 31, 2025, the outstanding number of shares of Common Stock of Pyxis Oncology was 62,264,215.

(Press release, Pyxis Oncology, NOV 3, 2025, View Source [SID1234659277])

On November 3, 2025 PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value, reported that new data from its ongoing Phase 1b clinical trial evaluating LYT-200, a first-in-class anti-galectin-9 monoclonal antibody, in relapsed/refractory acute myeloid leukemia (AML) will be shared on December 6th, 2025, during the 67th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando, Florida, by its Founded Entity Gallop Oncology. The accepted abstract reflects data as of July 8, 2025, and additional analyses based on a later data cut-off are expected to be presented during the ASH (Free ASH Whitepaper) meeting.

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!

The ongoing, open-label, dose-ranging trial is evaluating LYT-200 both as a monotherapy and in combination with the standard-of-care (SOC) regimen of venetoclax (VEN) and hypomethylating agents (HMA) in a very vulnerable population. All participants in the trial have previously been treated with SOC (median prior lines of treatment: 3; range: 1-7), and their disease had either returned or failed to respond.

The data submitted to ASH (Free ASH Whitepaper) reflect efficacy and safety findings for 31 participants in the monotherapy arm and 39 participants in the combination arm who received LYT-200 weekly at doses ≥7.5mg/kg. As a monotherapy, treatment with LYT-200 resulted in 1 marrow complete response (CR) and 3 partial responses (PRs). Notably, one PR in the monotherapy arm was maintained for 24 months as of the data cut off in an individual whose disease previously progressed following five prior rounds of treatment with SOC. When administered in combination with VEN/HMA, LYT-200 treatment resulted in 12 CRs, 1 PR, and 1 morphological leukemia-free state (MLFS). Importantly, CRs were achieved in this cohort across a diverse range of tumor subtypes, including KRAS, NRAS, HRAS, and JAK2 mutations, in patients who were previously fully refractory to SOC.

When evaluating patients with AML, a CR is the primary goal of treatment and means that no leukemia cells are detectable in the blood, fewer than 5% blasts remain in the bone marrow, and blood counts have returned to normal. Achieving a CR is generally associated with improved outcomes, including longer overall survival. A PR reflects a significant reduction in leukemia burden, with at least a 50% decrease in blasts, while an MLFS indicates that there are no leukemia cells visible and fewer than 5% blasts in the marrow, though blood counts have not yet recovered. While SOC in this advanced relapsed/refractory population typically achieves CR rates of 6-12% and median overall survival is less than 2.5 months,[1] LYT-200 has demonstrated a >30% CR rate in the combination cohort as of the data cut off, underscoring its potential to serve as a meaningful new treatment option.

Across all dose levels and treatment arms, LYT-200 was well tolerated. No dose-limiting toxicities were reported, and there were no LYT-200-related serious adverse events, discontinuations, or deaths. The most common adverse events potentially related to LYT-200 were mild and transient.

"The combination of this level of efficacy with a clean safety profile underscores the importance of advancing LYT-200 into its next phase of development, especially given the high relapse rates and poor survival outcomes in AML," said Luba Greenwood, JD, Chief Executive Officer of Gallop Oncology. "As survival data mature, we believe they could add another compelling dimension to LYT-200’s potential clinical profile for patients with relapsed/refractory AML, including those who have failed VEN/HMA or have mutations associated with poorer prognosis, where the need for new therapies remains urgent."

PureTech intends to share further matured data at ASH (Free ASH Whitepaper), including updated efficacy across dose levels, as well as survival and pharmacokinetic/pharmacodynamic data. Topline efficacy data are expected in the fourth quarter of 2025, with topline survival data anticipated in the first half of 2026. PureTech intends to engage with regulatory authorities to advance LYT-200 into a Phase 2 trial.

About AML and MDS

Acute myeloid leukemia (AML) is an aggressive blood cancer characterized by the rapid growth of abnormal myeloid cells in the bone marrow and blood. It is the most common form of acute leukemia in adults, with a five-year survival rate of less than 30%. Despite available therapies, many patients relapse or fail to respond, and outcomes are especially poor in the relapsed/refractory setting.

Myelodysplastic syndromes (MDS) are a group of rare blood cancers in which the bone marrow does not produce enough healthy blood cells. High-risk MDS often progresses to AML and is associated with limited treatment options and poor survival.

Together, AML and high-risk MDS represent areas of urgent unmet medical need where new therapies with improved efficacy and durability are critically needed. Importantly, the incidence of AML is increasing and the market is expected to grow to $6 billion by 2030, underscoring the scale of the opportunity to bring forward more effective therapies.

About LYT-200

LYT-200 is a fully human IgG4 monoclonal antibody targeting galectin-9, a key oncogenic driver and potent immunosuppressor in cancer. It is being developed for the potential treatment of hematological malignancies and solid tumors with otherwise poor survival rates. In an ongoing acute myeloid leukemia (AML) trial, LYT-200 has demonstrated clinical activity and disease stabilization in heavily pretreated patients, both as a monotherapy and in combination with standard-of-care therapy.

LYT-200 has been granted Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia, underscoring the high unmet need in this disease and the potential for LYT-200 to serve as a meaningful therapeutic option.

(Press release, PureTech Health, NOV 3, 2025, View Source [SID1234659276])

On November 3, 2025 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for high unmet need diseases, reported financial results for the third quarter ended September 30, 2025, and provided a business update.

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!

"Throughout the third quarter, we made strong progress across our gene editing pipeline and reported compelling Phase 1 safety and efficacy data for PBGENE-HBV at the International Coalition to Eliminate HBV Cure Symposium. We’re also eagerly awaiting a late-breaking oral presentation at AASLD on November 10th," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "The PBGENE-HBV data presented so far this year has shown proof of durable activity and a safety profile that allows us to continue dose escalation in pursuit of achieving a complete cure for hepatitis B patients. With great excitement, in 2026 we anticipate starting the first-in-human clinical trial with our second program, PBGENE-DMD for DMD patients, following our targeted IND submission by the end of 2025. We are highly encouraged by the unique preclinical data showing the potential to restore a nearly full length dystrophin gene which is native to the human body with the goal of improving function over time."

Wholly-Owned Portfolio:

PBGENE-HBV (Hepatitis B Viral Elimination Program):

On October 14th, Precision announced that it had been selected to deliver a late-breaking oral presentation at the upcoming Liver Meeting 2025 during the 75th American Association for the Study of Liver Diseases. The oral presentation will feature new data from the multiple cohorts of the ongoing Phase 1 ELIMINATE-B Trial.

On October 7th, Precision announced that the first clinical trial sites in the U.S. for the ELIMINATE-B trial had officially been activated. The site at Massachusetts General Hospital in Boston, Massachusetts, is now actively recruiting chronic hepatitis B patients along with multiple global clinical trial sites.

On September 12th, the Company presented data from the Phase 1 ELIMINATE-B trial of PBGENE-HBV at the 6th International Coalition to Eliminate HBV Cure Symposium in Berlin, Germany. To date, PBGENE-HBV has shown to be well-tolerated by patients in both Cohort 1 and Cohort 2, who received multiple doses of 0.2 mg/kg of PBGENE-HBV and 0.4 mg/kg, respectively. In addition, PBGENE-HBV has demonstrated a substantial HBsAg reduction in all patients across Cohort 1 with one patient in Cohort 1 achieving a durable HBsAg reduction of approximately 50% from baseline that was ongoing seven months following initial dose administration. Given the favorable safety profile of Cohorts 1 and 2, the Data Monitoring Committee recommended the Company to proceed with dosing Cohort 3 which occurred during the third quarter of 2025.

On September 8th, Precision announced the issuance of a U.S. Patent (No. 12,410,418) by the U.S. Patent and Trademark Office (USPTO) titled "Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B Virus genome." The composition of matter claims in the patent encompass the PBGENE-HBV ARCUS nuclease which has an expiration date in March 2042. Patents in Europe and Hong Kong granted earlier this year included similar composition of matter claims.

PBGENE-DMD (Muscle Targeted Excision Program):

On October 10th, the Company presented a late-breaking poster presentation at the 30th Annual International Congress of the World Muscle Society meeting highlighting durable improvements in muscle function over time through increased dystrophin expression and dystrophin-positive cells for PBGENE-DMD. The data from a DMD mouse model demonstrated that dystrophin protein was detected in all muscles evaluated following the administration of PBGENE-DMD at doses up to 1×1014 vg/kg, with increased expression observed at nine months versus prior timepoints in the quadriceps, gastrocnemius, heart, and diaphragm, resulting in substantial and sustained functional muscle improvement. An increase in dystrophin-positive muscle cells were observed in all muscles. The maximum force output was significantly improved over untreated DMD mice at three, six and nine months post-treatment, highlighting strong durability of PBGENE-DMD outcomes.

Precision has completed final toxicology studies and is manufacturing clinical supplies, with an anticipated IND filing by the end of 2025. Pending IND clearance, Phase 1 initiation in DMD patients is anticipated in the first half of 2026 with initial data expected to follow in the second half of 2026.

Partnered In Vivo Gene Editing Programs:

iECURE-OTC (Gene Insertion Program)

Led by partner, iECURE, ECUR-506 is an ARCUS-mediated in vivo targeted gene insertion program currently in a first-in-human trial (OTC-HOPE) evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency.

Several recent medical conference presentations with updated ECUR-506 clinical data include:

The 6th International Symposium on Urea Cycle Disorders and the 15th International Congress of Inborn Errors of Metabolism, both held in early September in Kyoto, Japan. In October, presentations at medical conferences included the European Society of Gene & Cell Therapy Annual Congress held in Sevilla, Spain, and the American Society of Human Genetics Annual Meeting in Boston, Massachusetts.

These data presentations build upon previously reported clinical results demonstrating complete clinical response in the first participant at the lowest dose level (1.3×1013 GC/kg) of ECUR-506, as defined by the study protocol. The OTC-HOPE study is ongoing in the U.K., the U.S., Australia, and Spain with data from the trial expected in the first half of 2026.

Non-Core Ex Vivo Programs:

Azer-Cel (azercabtagene zapreleucel allogeneic CAR T treatment for cancer)

Imugene Limited, Precision’s clinical stage partner developing azer-cel for oncology indications, announced on September 17th, additional efficacy data from its Phase 1b clinical trial evaluating azer-cel in patients with relapsed/refractory diffuse large B-cell lymphoma. The updated data showcased an overall response rate of 81% in patients treated with azer-cel and IL-2 with seven complete responses and six partial responses including several patients remaining in durable remission beyond one year.

On October 28th, Imugene announced the first efficacy results from the CAR T-naïve indication cohort of its ongoing Phase 1b trial of azer-cel. Of the six evaluable CAR T-naïve patients, five (83%) achieved an overall response including three (50%) complete responses. The result of the sixth patient’s follow-up scan is pending. A total of ten patients have been treated in this CAR T-naïve cohort thus far, with additional results to come upon patient follow-up. These initial results encompass several rare lymphoma subtypes, notably Waldenström Macroglobulinemia (WM), Marginal Zone Lymphoma (MZL) and Primary Central Nervous System Lymphoma (PCNSL).

Imugene is actively enrolling patients in the Phase 1b azer-cel trial at ten U.S. sites and five sites in Australia. Imugene has scheduled a Type C meeting with the U.S. Food and Drug Administration (FDA) to discuss potential pivotal study design options for azer-cel. The decision to proceed to a meeting with the FDA to discuss a pivotal trial reflects the positive, durable clinical data that has been generated to date and adds to the growing clinical data set supporting the ARCUS platform. On October 31, 2025, Precision received an $8 million milestone payment in cash and stock from Imugene.

Corporate Updates:

Mark Sulkowski, M.D. Appointed Head Clinical Development Advisor

In August, Mark Sulkowski, Professor of Medicine at the Johns Hopkins University School of Medicine and renowned expert in hepatic and infectious diseases has expanded his advisory role with Precision. In the newly created role, Head Clinical Development Advisor, Dr. Sulkowski will work closely with Precision’s leadership and cross-functional teams to support clinical strategy across the development lifecycle for the Company’s on-going PBGENE-HBV Phase 1 clinical trial as well as initiation of later stage trials. His advisory role will focus on optimizing clinical trials, including translational integration, and aligning scientific rationale with regulatory objectives.

Quarter Ended September 30, 2025 Financial Results

In July 2025, the Company implemented operating efficiencies, including employment related and other expense reductions, to reduce annual operating expenses and extend its expected cash runway. In the third quarter of 2025, the Company realized reductions in early research and general & administrative expenses which reduced the Company’s operating expenses compared to both the second quarter of 2025 and third quarter of 2024.

Cash, Cash Equivalents, and Restricted Cash: As of September 30, 2025, Precision had approximately $71.2 million in cash, cash equivalents and restricted cash. Based on its expected cash runway, Precision believes it is sufficiently capitalized to reach important milestones for PBGENE-HBV and PBGENE-DMD. The Company expects existing cash and cash equivalents, potential near-term cash from CAR T transactions, along with expected operating efficiencies, operational receipts, and availability of Precision’s at-the-market (ATM) facility to extend Precision’s cash runway into the second half of 2027.

Revenues: Total revenues for the quarter ended September 30, 2025, were less than $0.1 million as compared to $0.6 million for the quarter ended September 30, 2024. The decrease was primarily the result of less billable effort under the Novartis Agreement.

Research and Development Expenses: Research and development expenses were $13.4 million for the quarter ended September 30, 2025, as compared to $13.1 million for the quarter ended September 30, 2024. The increase was primarily the result of an increase in the PBGENE-DMD program partially offset by decreases in the PBGENE-HBV program as it transitioned to the clinic at the end of 2024 and the PBGENE-3243 program which has been paused.

General and Administrative Expenses: General and administrative expenses were $7.3 million for the quarter ended September 30, 2025, as compared to $8.8 million for the quarter ended September 30, 2024. The decrease was primarily the result of employee-related costs and other general and administrative expenses.

Net Loss: Net loss was $21.8 million, or ($1.84) per share (basic and diluted), for the quarter ended September 30, 2025. Net loss was $16.4 million or $(2.25) per share (basic and diluted) for the quarter ended September 30, 2024.

About PBGENE-HBV, A Viral Elimination Program

PBGENE-HBV is Precision’s wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to be a potentially curative treatment for chronic Hepatitis B infection. PBGENE-HBV is the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate the root cause of chronic Hepatitis B, cccDNA, while inactivating integrated HBV DNA. The ELIMINATE-B trial is investigating PBGENE-HBV at multiple ascending dose levels with three dose administrations per dose level in patients with chronic Hepatitis B. PBGENE-HBV has been granted Breakthrough Therapy designation by the FDA.

About PBGENE-DMD, A Muscle-Targeted Excision Program

PBGENE-DMD is Precision’s development program for the treatment of DMD. DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the U.S. alone. There are currently no approved therapies that can drive durable and significant functional improvements over time. PBGENE-DMD is designed to improve function for more than 60% of patients afflicted with DMD by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene. The aim of this approach is to restore a near-full length functional dystrophin protein within the body that more closely resembles normal dystrophin as opposed to synthetic, truncated dystrophin approaches with minimal functional benefit. PBGENE-DMD has received both Rare Pediatric Disease and Orphan Drug designations from the U.S. FDA.

(Press release, Precision Biosciences, NOV 3, 2025, View Source [SID1234659275])