On December 10, 2023 Antengene Corporation Limited ("Antengene" SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class medicines for cancer, reported that the first patient has been dosed in the Phase I PERFORM trial of ATG-031 for the treatment of patients with advanced solid tumors or B-cell non-Hodgkin’s lymphoma (B-NHL), at The University of Texas MD Anderson Cancer Center, the lead center of the study that also includes three other clinical trial centers across the U.S (Press release, Antengene, DEC 10, 2023, View Source [SID1234638385]).

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The PERFORM trial is a first-in-human, multi-center, open-label, Phase I dose-finding study of ATG-031 in patients with advanced solid tumors or B-NHL. The study’s primary objective is to evaluate the safety and tolerability of ATG-031 as a monotherapy, and determine the appropriate dose for Phase II studies. The secondary objective is to characterize the pharmacology, evaluate the immunogenicity, and assess the preliminary efficacy of ATG-031.

"We are delighted that the PERFORM trial has successfully dosed its first patient in the U.S. CD24 is a target with great potential in regulating macrophage activities in tumor environment. Supported by the striking preclinical efficacy demonstrated by blocking CD24 in preclinical studies and a promising preclinical safety profile based on CD24’s limited expression in normal tissue, Antengene has quickly initiated the PERFORM study in the U.S. to further assess the safety and tolerability of ATG-031 in late stage cancer patients," said Dr. Amily Zhang, Antengene’s Chief Medical Officer. "Moving forward, we will continue to work closely with investigators at MD Anderson and three other clinical trial centers in the U.S. to bring clinical benefit to more patients as soon as possible."

"With the joint persistent efforts from Antengene’s internal teams, we have made great strides to the development of ATG-031, a first-in-class anti-CD24 monoclonal antibody targeting the novel ‘don’t eat me’ pathway, while making rapid progress with the clinical development of other core assets. To date, we have established close collaboration with more than 100 clinical trial centers in the U.S., Australia, and the Mainland of China," said Dr. Jay Mei, Antengene’s Founder, Chairman and CEO. "Remaining committed to our global innovation strategies and steadily expediting our clinical programs, we will focus on next generation novel treatments to address broad unmet medical needs post the current immune checkpoint inhibitors."

About ATG-031
ATG-031 is a first-in-class humanized CD24 monoclonal antibody which inhibits the "don’t eat me" signal and enhances macrophage-mediated phagocytosis of cancer cells. Tumor cells evade the surveillance of the human immune system by over-expressing "don’t eat me" surface proteins that signal macrophages to prevent the detection and phagocytosis of cancer cells. CD24 (cluster of differentiation 24) is a prominent "don’t eat me" signal that plays a significant role in tumor immune evasion by suppressing macrophage-mediated phagocytosis. Compared to CD47, another well-known "don’t eat me" target, CD24 has a more restricted distribution in normal tissue and higher expression in cancerous tissue. In addition，unlike CD47，CD24 is not expressed on human red blood cells，allowing for a wider therapeutic window and minimal on-target-off-tumor toxicity as a CD24-targeted therapy.

As a novel innate immune checkpoint, CD24 orchestrates immune evasion through its interaction with the inhibitory receptor Siglec-10 (sialic-acid-binding Ig-like lectin 10) expresses on tumor-associated macrophages (TAMs). Preclinical data presented in 2023 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (AACR 2023) demonstrated that ATG-031 can specifically bind to CD24 with nM affinity and block the interaction of CD24 and Siglec-10. Furthermore, ATG-031 induces efficient phagocytosis with a picomolar EC50 and stimulates the pro-inflammatory cytokines production by macrophages.

On December 10, 2023 GlycoMimetics, Inc. (Nasdaq: GLYC), a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers and inflammatory diseases, reported that the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting will feature two presentations on uproleselan, including an investigator-initiated trial (IIT) studying the first-in-class E-selectin antagonist in patients with treated secondary Acute Myeloid Leukemia (ts-AML) (Press release, GlycoMimetics, DEC 10, 2023, View Source [SID1234638370]).

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"We are excited by the results from this IIT demonstrating the safety and preliminary efficacy of uproleselan in ts-AML patients, a group with few treatment options and persistent high unmet need," said Edwin Rock, M.D., Ph.D., Chief Medical Officer of GlycoMimetics. "These findings underscore the broad potential of uproleselan, if successfully developed in combination with existing therapies, to benefit people with heterogeneous forms of AML. We are committed to exploring this potential across different age groups and disease settings through studies conducted by partners and independent investigators, and we thank them for their continued collaboration."

Uproleselan Added to Cladribine Plus Low Dose Cytarabine (LDAC) in Patients with Treated Secondary Acute Myeloid Leukemia (TS-AML)

Presenter: Emmanuel Almanza Huante, M.D., Department of Leukemia
The University of Texas MD Anderson Cancer Center, Houston, TX
Session: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster II
Time and Location: Sunday, December 10, 2023: 6:00 PM-8:00 PM PST, Halls G-H, San Diego Convention Center
Poster Number: 2922

This Phase 1b/2 clinical trial evaluated safety, tolerability, and preliminary efficacy of uproleselan added to cladribine and LDAC in patients with ts-AML.
This rare, high-risk study population is defined by prior chemotherapy treatment of a previous hematologic disorder, such as myelodysplastic syndrome. Median survival of ts-AML patients is less than 5 months.
A majority of the 20 patients enrolled in this IIT were male (n=14), and median age of enrolled patients was 72 years. All patients had unfavorable cytogenetics and had previously received treatment with a hypomethylating agent. Also, 11 patients (55%) had received prior treatment with venetoclax, and five (25%) had undergone stem cell transplantation.
At a median follow-up of 8.1 months, results from 18 evaluable patients showed that cladribine and LDAC combined with uproleselan generated few treatment-related adverse events. The combination produced an overall response in 7 (39%) patients and reduced bone marrow blasts (morphologic leukemia-free state) in 13 (72%) patients. Median overall survival was 5.3 months.
Disease responses were seen irrespective of previous hypomethylating agent and venetoclax exposure. Three patients went on to receive a potentially curative hematopoietic cell transplantation (HCT). Study investigators concluded these data support this low-risk approach to marrow blast reduction and disease control in preparation for HCT.
Pediatric Acute Leukemia (PedAL) Screening Trial – Developing New Therapies for Relapsed Leukemias (APAL2020SC)

Presenter: Michele S. Redell, MD, Texas Children’s Cancer Center, Baylor College of Medicine, Houston
Session: 613. Acute Myeloid Leukemias: Clinical and Epidemiological: Poster I
Time and Location: Saturday, December 9, 2023, 5:30 PM-7:30 PM PST, Halls G-H, San Diego Convention Center
Poster Number: 1492

This poster reports progress to date of the PedAL Initiative, a screening trial (APAL2020SC/NCT0472624) sponsored by the Leukemia & Lymphoma Society in North America, Australia and New Zealand. Primary aims of PedAL are (1) to use clinical and biological characteristics to screen for eligibility for therapeutic sub-trials; and (2) to maintain a longitudinal data registry and biobank for relapsed leukemia. As a screening protocol, PedAL serves as the entry point for associated therapeutic sub-trials.
One participating sub-trial is a National Cancer Institute sponsored Phase 1 dose escalation study (APAL2020E/PEPN2113), which assesses safety and preliminary activity of uproleselan plus fludarabine and high dose cytarabine in pediatric AML patients after 2 or more prior therapies. Expected enrollment of 18 patients has begun with the first patient receiving study therapy in late 2023.
About Uproleselan

Discovered and developed by GlycoMimetics, uproleselan is an investigational, first-in-class E-selectin antagonist. Uproleselan (yoo’ pro le’se lan), currently in a broad development program including a late-stage Phase 3 trial in acute myeloid leukemia (AML), has received Breakthrough Therapy and Fast Track designations from the U.S. Food and Drug Administration and Breakthrough Therapy designation from the Chinese National Medical Products Administration for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin binding and stimulation of myeloid cells. E-selectin is expressed on the surface of blood vessels, and its binding to myeloid cells confers a pro-survival effect via NF-kB signaling. Uproleselan is being developed to provide a novel approach to disrupting established mechanisms of leukemic cell resistance.

On December 10, 2023 Taiho Oncology, Inc. reported results of a U.S. real-world study of oral decitabine and cedazuridine (DEC-C) in patients with myelodysplastic syndromes (MDS), a rare form of blood cancer (Press release, Taiho, DEC 10, 2023, View Source [SID1234638386]). Results of the retrospective real-world analysis of use patterns for hypomethylating agents suggest oral DEC-C as a treatment option with the potential to reduce patient and caregiver burden, while maintaining patients on therapy. Data from the study were shared during an oral presentation (Abstract #548) at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition.

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"For patients with myelodysplastic syndromes, therapy with intravenous or subcutaneous hypomethylating agents has been associated with increased patient burden, which can adversely affect treatment compliance and, subsequently, outcomes," said Amer Zeidan, MBBS, MHS, Associate Professor of Medicine (Hematology), Yale School of Medicine, Interim Chief, Hematologic Malignancies, Yale Cancer Center and Smilow Cancer Hospital, and study investigator. "Previous studies have shown that oral decitabine and cedazuridine provides comparable safety and efficacy to parenteral HMAs,1,2,3 and what we found in this real-world study is that this oral therapy resulted in a trend for improved persistence as treatment extended beyond the first six months. Based on these results and the potential for reduced treatment burden, oral decitabine and cedazuridine may be a viable alternative to intravenous or subcutaneous HMAs."

In this study of 1,569 patients, 160 received oral DEC-C and 1,409 received intravenous/subcutaneous (IV/SC) HMAs. After matching, there were 158 patients in each treatment cohort. Longitudinal persistence – the accumulation of time from initiation to discontinuation of therapy4 – was comparable between the matched oral DEC-C and intravenous/subcutaneous HMA cohorts during the first 6 months post-index, with similar proportions receiving the maximum number of treatment cycles (based on a 28-day cycle) at each month following index (73.8% vs 71.1%, 46.5% vs 48.7% and 28.6% vs 24.8%, for 2, 4 and 6 months, respectively). However, a trend toward improved persistence with oral DEC-C versus intravenous/subcutaneous HMAs was observed in patients receiving treatment beyond 6 months (25.0% vs 17.0%, 18.5% vs 9.0% and 11.4% vs 7.6% for 8, 10 and 12 months, respectively). Mean time to discontinuation of treatment was numerically higher for the oral DEC-C users compared with the IV/SC HMA group (87.7 vs 82.0 days); however, the differences were not statistically significant.

"For many patients living with MDS, the potential for additional costs and time associated with travel for regular infusions can be a significant burden during a stressful time in their lives," said Tehseen Salimi, MD, MHA, Senior Vice President Medical Affairs, Taiho Oncology. "Our goal with this study was to measure the real-world impact of oral therapy. As a leader in the development of orally administered anti-cancer agents, Taiho Oncology is pleased to see how outcomes of this real-world study show some of the potential benefits of an oral therapy in myelodysplastic syndromes."

About the Study

This study was a retrospective analysis using the U.S. Cerner Enviza claims database; medical and prescription claims data for patients were linked to mortality data from Datavant. Adults aged ≥18 years, who were diagnosed with MDS and who had ≥1 claim for an HMA (oral DEC-C or IV/SC HMA) were included. Patients had variable follow-up after the index date and were followed until end of enrollment, death or end of study. Longitudinal persistence was assessed according to the number of cycles of therapy received during follow-up, where a cycle was defined as either 3-10 days of administration of index intravenous/subcutaneous HMA or one claim for oral DEC-C within a 28-day cycle.

About Myelodysplastic Syndromes
Myelodysplastic syndromes are a heterogeneous group of hematopoietic stem cell disorders characterized by dysplastic changes in myeloid, erythroid, and megakaryocytic progenitor cells, and associated with cytopenias affecting one or more of the three lineages. In the U.S., approximately 20,000 cases of MDS are reported every year,5 with an overall age-adjusted incidence rate of 4.0 cases per 100,000 population.6 MDS may progress into acute myeloid leukemia (AML) in approximately 30-40% of patients.7,8 The prognosis for MDS patients is poor; patients die from complications associated with cytopenias (infections and bleeding) or from transformation to AML.

On December 10, 2023 ImmunoGen Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported new safety and efficacy findings from the newly diagnosed (ND) cohorts of the Phase 1b/2 study of pivekimab sunirine (pivekimab) in combination with azacitidine (Vidaza) and venetoclax (Venclexta), (pivekimab triplet) in patients with ND acute myeloid leukemia (AML) (Press release, ImmunoGen, DEC 10, 2023, View Source [SID1234638371]). These findings will be presented in a poster session at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, California.

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"We are pleased to share these new findings at ASH (Free ASH Whitepaper), which demonstrate encouraging anti-leukemia activity of the pivekimab triplet in newly diagnosed AML, a disease in which long-term survival unfortunately remains limited," said Naval Daver, MD, Associate Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. "The MRD negativity rates, which are indicative of a deep remission, are particularly promising in the treated patient population. This encouraging activity, along with a manageable safety profile, support the continued evaluation of this novel triplet in this setting."

PIVEKIMAB SUNIRINE, A CD123-TARGETING ANTIBODY-DRUG CONJUGATE, IN COMBINATION WITH AZACITIDINE AND VENETOCLAX IN PATIENTS WITH NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA
Lead Author: Navel Daver, MD
Poster Session: 616 (Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster II)
Date and Time: Sunday, December 10, 2023, 6:00-8:00 p.m. PT / 9:00-11:00 p.m. ET
Publication Number: 2906

In the open-label, multicenter, Phase 1b/2 study of pivekimab in combination with azacitidine and venetoclax in patients with ND CD123-positive AML, patients received the recommended Phase 2 dose of pivekimab at 0.045 mg/kg on day 7, azacitidine at 75 mg/m2 daily on days 1-7, and venetoclax at up to 400 mg for at least 14 days or up to 28 days, based on cohort assignment, in a 28-day cycle. The primary endpoints are complete remission (CR) rate, composite CR rate (CCR [CR+CRh+CRp+CRi]), minimal residual disease (MRD) negativity rate, and duration of remission. Key secondary endpoints are safety, pharmacokinetics, and immunogenicity.

Key findings for 50 ND patients (n=25 per cohort) as of September 29, 2023 (data cut-off) include:

Anti-Leukemia Activity

In the overall population, CCR rate was 68% (34/50), CR rate was 54% (27/50), and MRD negativity rate among evaluable patients achieving CCR was 76% (22/29). MRD was assessed centrally by flow cytometry with <0.1% considered negative. Response rates and MRD negativity were numerically comparable between cohorts 1 and 2, despite differences in the venetoclax schedule.
In a post hoc subset analysis of patients unfit for intensive chemotherapy (i.e. patients >75 years of age, and/or with pre-specified comorbidities) (n=23), CCR rate was 78% (18/23), CR rate was 61% (14/23), and MRD negativity rate was 79% (11/14).
In patients known to be TP53wt (n=25), CCR rate was 88% (22/25), CR rate was 84% (21/25), and MRD negativity rate was 80% (16/20). CCR and MRD negativity rates, respectively, were high across other major molecular subsets, including:
FLT3 (ITD or TKD): 100% (6/6) and 100% (6/6)
IDH1 mutant: 100% (4/4) and 67% (2/3)
IDH2 mutant: 100% (6/6) and 83% (5/6)
NPM1 mutant: 100% (8/8) and 86% (6/7)
K/NRAS mutant: 50% (3/6) and 67% (2/3)
TP53 mutant: 50% (7/14) and 50% (3/6)
Among all MRD negative patients, the median time to MRD negativity was 1.87 months (range: 0.79-5.16 months).
Although follow-up duration was short (median 5.2 months), landmark overall survival estimate at 6 months is 86%.
The study is continuing to enroll newly diagnosed unfit AML patients.
Safety

The triplet displayed a manageable safety profile; no new safety signals were observed compared to previously reported data.
The most common non-hematologic treatment-emergent adverse events (TEAEs) (all grades [grade 3+]) seen in ≥20% of all patients were constipation (48% [2%]), peripheral edema (44% [4%]), diarrhea (40% [2%]), hypophosphatemia (34% [2%]), nausea (32% [4%]), hypokalemia (28% [4%]), fatigue (24% [6%]), hypotension (24% [2%]), and pyrexia (24% [0%]). In the overall population:
Rates of cytopenias were similar to those observed with azacitidine and venetoclax, with a median neutrophil recovery to ≥500/µL and platelet recovery to ≥50,000/µL by day 34 and day 22, respectively.
No veno-occlusive disease, capillary leak syndrome, or sinusoidal obstruction syndrome were observed.
Infusion-related reactions (IRRs) occurred in 16% of patients (0 grade 3+ IRRs).
Discontinuations due to an adverse event (AE) were 4% (2 patients).
30-day mortality was 0%.
60-day mortality was 4% (2 patients; due to pneumonia and early disease progression).
"Building upon our initial findings in frontline AML presented last year, these data show broad and consistent response rates in a larger study population and across major molecular subsets of interest, including those patients with biological mutations making them high-risk," said Michael Vasconcelles, MD, ImmunoGen’s Executive Vice President, Research, Development, and Medical Affairs. "We are pleased with the low early mortality and manageable safety profile observed, in particular the lack of prolonged cytopenias. We look forward to continuing to expand our cohort of newly diagnosed unfit patients to inform the development path for pivekimab in AML."

PRECLINICAL POSTERS
ImmunoGen is also presenting two preclinical posters at ASH (Free ASH Whitepaper).

Title: Venetoclax Synergizes with IMGN632, a Novel CD123-Targeting Antibody Conjugated to a DNA Alkylating Payload, By Suppressing DNA Damage Response and Potentiating Apoptosis in Acute Myeloid Leukemia in Vitro Models
Presenter: Anna Skwarska
Session: 604 (Molecular Pharmacology and Drug Resistance: Myeloid Neoplasms: Poster III)
Date and Time: Monday, December 11, 2023, 6:00-8:00 p.m. PT / 9:00-11:00 p.m. ET
Publication Number: 4155

Title: Spatial Response to Pivekimab Sunirine In Vivo in a BPDCN Model
Presenter: Margaux Poussard
Session: 604 (Molecular Pharmacology and Drug Resistance: Myeloid Neoplasms: Poster II)
Date and Time: Sunday, December 10, 2023, 6:00-8:00 p.m. PT / 9:00-11:00 p.m. ET
Publication Number: 2791

Additional information can be found at View Source, including abstracts.

ABOUT PIVEKIMAB SUNIRINE
Pivekimab sunirine is a CD123-targeting ADC in clinical development for hematological malignancies, including blastic plasmacytoid dendritic cell neoplasm (BPDCN), acute myeloid leukemia (AML), and other CD123+ hematologic malignancies. Pivekimab is currently being evaluated as monotherapy for patients with BPDCN and in combination with azacitidine (Vidaza) and venetoclax (Venclexta) for patients with untreated and relapsed/refractory AML. Pivekimab uses one of ImmunoGen’s novel indolinobenzodiazepine (IGN) payloads, which alkylate DNA and cause single-strand breaks without crosslinking. IGNs are designed to have high potency against tumor cells, while demonstrating less toxicity to normal marrow progenitors than other DNA-targeting payloads. The European Medicines Agency (EMA) granted orphan drug designation to pivekimab for the treatment of BPDCN in June 2020. Pivekimab also holds this designation in the US. In October 2020, the FDA granted pivekimab Breakthrough Therapy designation in relapsed/refractory BPDCN.

ABOUT ACUTE MYELOID LEUKEMIA (AML)
AML is a cancer of the bone marrow cells that produce white blood cells. It causes the marrow to increasingly generate abnormal, immature white blood cells (blasts) that do not mature into effective infection-fighting cells. The blasts quickly fill the bone marrow, impacting the production of normal platelets and red blood cells. The resulting deficiencies in normal blood cells leave the patient vulnerable to infections, bleeding problems, and anemia. It is estimated that, in the US alone, more than 20,000 people will be diagnosed with AML and more than 11,000 will die from the disease this year.

ABOUT CD123
CD123, the interleukin-3 alpha chain, is expressed on multiple myeloid and lymphoid cancers including AML, BPDCN, ALL, chronic myeloid leukemia, and myeloproliferative neoplasms. With limited expression on normal hematopoietic cells, rapid internalization, and expression on AML leukemia stem cells, CD123 is a clinically validated therapeutic target.

On December 10, 2023 Remedy Plan Therapeutics ("Remedy Plan"), a small molecule therapeutics start-up company transforming the field of NAMPT inhibition, reported new data to be shared during an oral presentation at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in San Diego, California, December 9-12th (Press release, Remedy Plan, DEC 10, 2023, View Source [SID1234638387]).

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Remedy Plan will present data on RPT1G, a clinical drug candidate for the treatment of solid and hematological malignancies. RPT1G is a small molecule NAMPT inhibitor with a unique, first-in-class mechanism of action that is optimized to preserve the cellular metabolism in healthy cells, while inhibiting activity in malignant cells. This is achieved through hyperbolic inhibition of NAMPT that is fractional and highly tunable. Preclinical and animal studies revealed that RPT1G is efficacious in acute leukemia, lymphoma, and solid tumor models, selectively killing cancer cells. Importantly, RPT1G was thousands-fold better tolerated by healthy cells, without the severe on-target toxicities reported for other NAMPT inhibitors.

"We are excited to introduce our clinical candidate RPT1G, a first-in-class NAMPT drug that shows efficacy without the severe on-target toxicities seen with other NAMPT inhibitors," said Greg Crimmins, Ph.D., CEO and Founder of Remedy Plan. "NAMPT dysregulation is responsible for more than 20 diseases, but efforts to target NAMPT to date have been hampered by on-target toxicities. Our groundbreaking mechanism of action has the potential to revolutionize the field of NAMPT inhibition, unlocking opportunities for treating hematological malignancies and solid tumors, as well as autoimmune and metabolic diseases."

NAMPT is central to cancer metabolism and is up-regulated in many solid and hematological malignancies, making it a high-value oncology target. Based on the promising data presented at ASH (Free ASH Whitepaper), RPT1G will be advancing to the clinic in 2024.

Abstract and Presentation details:

Remedy Plan’s CEO and Founder Greg Crimmins, Ph.D. will present the data on Sunday December 10th at 10:30AM PST in Grand Hall B of the Manchester Grand Hyatt San Diego, and all meeting participants are encouraged to attend either in person or online.

A 1st-in-Class Small Molecule NAMPT Inhibitor as a Novel Therapeutic for Acute Lymphocytic Leukemia. Abstract #419, Oral Session: 605, Molecular Pharmacology and Drug Resistance: Lymphoid Neoplasms: Targeted Therapy in Lymphoid Leukemias. The full abstract is available here.