Kura Oncology Reports Preclinical Data Showing Ability of KO-2806 to Enhance Antitumor Activity of KRAS?¹²? Inhibitors in Non-Small Cell Lung Cancer

On September 28, 2023 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported preclinical data supporting the development of its next-generation farnesyl transferase inhibitor (FTI) KO-2806 in combination with KRASG12C inhibitors to drive tumor regressions and durable responses in KRASG12C-mutant non-small cell lung cancer (NSCLC) (Press release, Kura Oncology, SEP 28, 2023, View Source [SID1234635501]).

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The new findings are being presented in an oral session today at the 5th RAS-Targeted Drug Development Summit in Boston. A copy of the presentation, entitled "Farnesyl Transferase Inhibitors – Evolution from Targeting HRAS to Overcoming Adaptive Resistance to Targeted Therapies," is available in the Posters and Presentations section on Kura’s website.

KRASG12C inhibitors have previously been shown to activate RTK signaling, leading to ERK-RSK and/or mTOR-S6 pathway reactivation. The Company’s new preclinical data show that co-treatment of preclinical models of KRASG12C-mutant NSCLC with KO-2806 and adagrasib deepens signaling inhibition at multiple nodes, including the MAPK and mTOR pathways, while decreasing cell proliferation. In both cell-derived (CDX) and patient-derived (PDX) xenograft models originating from NSCLC tumors, the combination of KO-2806 with adagrasib induced tumor regressions. In addition, the CDX and PDX models demonstrated enhanced duration and depth of antitumor response compared to adagrasib as a single-agent therapy.

"Despite advances with KRAS-targeted therapies, a significant unmet need remains for patients with KRASG12C-mutant NSCLC as acquired resistance occurs early and often," said Francis Burrows, Ph.D., Senior Vice President, Translational Research. "We are highly encouraged by these first preclinical data for KO-2806, which demonstrate the potential for FTIs as a mechanism-based combination agent to enhance antitumor activity of targeted therapies, such as KRASG12C inhibitors."

Kura is on track to dose the first patient in its Phase 1 dose-escalation trial of KO-2806 (FIT-001) in the second half of 2023. Concurrent with the monotherapy dose escalation, the Company plans to evaluate KO-2806 in dose-escalation combination cohorts with other targeted therapies in advanced solid tumors, including clear cell renal cell carcinoma (ccRCC) and KRASG12C NSCLC.

IMUNON Reports Interim Progression-Free Survival and Overall Survival Data in Phase 1/2 OVATION 2 Study in Advanced Ovarian Cancer

On September 28, 2023 IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage biotechnology company focused on developing DNA-mediated immunotherapies and next-generation vaccines, reported interim progression-free survival (PFS) and overall survival (OS) data with IMNN-001 in its Phase 1/2 OVATION 2 Study (Press release, IMUNON, SEP 28, 2023, View Source [SID1234635500]). IMNN-001 is the Company’s IL-12 gene-mediated immunotherapy based on its TheraPlas technology. Full enrollment of 110 patients was reached in September 2022.

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OVATION 2 is evaluating the dosing, safety, efficacy and biological activity of intraperitoneal IMNN-001 in combination with neoadjuvant chemotherapy (NACT) in patients newly diagnosed with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer. NACT is designed to shrink the tumors as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three additional cycles of chemotherapy to treat any residual tumor.

As expected for a Phase 1/2 study, the study is directional and was designed with an 80% confidence interval to show an approximate 33% improvement in PFS, when comparing the treatment arm (NACT + IMNN-001) with the control arm (NACT only). The secondary endpoints include OS, objective response rate (ORR), pathological response, surgical response and serologic response. The study was not powered for p values of 0.05. The final readout of this study is expected by mid-2024. A positive readout would inform next development steps.

Interim data from the intent-to-treat (ITT) population being reported today show efficacy trends in PFS, demonstrating a delay in disease progression in the treatment arm of approximately 33% compared with the control arm, with the hazard ratio nearing the required value. Preliminary OS data follows a similar trend, showing an approximate 9-month improvement in the treatment arm over the control arm.

Subgroup analyses show patients treated with a PARP inhibitor (PARPi) as maintenance therapy had longer PFS and OS if they were also treated with IMNN-001 compared with patients treated with NACT only. This was not a pre-specified subgroup as PARP inhibitors were approved after the OVATION 2 Study was initiated.

The median PFS in the PARPi + NACT group and the PARPi + NACT + IMNN-001 group was 15.7 months and 23.7 months, respectively.
The median OS in the PARPi + NACT group was 45.6 months and has not yet been reached in the PARPi + NACT + IMNN-001 group.
While the data is still preliminary, the Company has concluded at this point that patients treated with a combination of NACT + PARPi + IMNN-001 appear to have the greatest benefit and should be the focus of on-going follow up.

IMUNON also continues to see benefits in other secondary endpoints including an approximately 20% higher R0 tumor resection score and a doubling of the CRS 3 chemotherapy response score to approximately 30% in the treatment arm versus 14% in the control arm. A complete tumor resection (R0) is a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed. Chemotherapy response score is considered a good prognostic indicator in ovarian cancer. Safety analyses continue to show good tolerability of IMNN-001 in this setting.

Commenting on the interim data, Dr. Corinne Le Goff, IMUNON’s president and chief executive officer, said, "We are encouraged by these interim results and are particularly intrigued by the overall survival trends in the subgroup of patients who received PARP inhibitors, neoadjuvant chemotherapy and IMNN-001. While the number of patients in this subgroup is relatively small, this regimen may hold potential in treatment strategies as we continue to monitor patients enrolled in OVATION 2, with expectations to report topline results in mid-2024."

About IMNN-001 Immunotherapy

Designed using IMUNON’s proprietary TheraPlas platform technology, IMNN-001 is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system that enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anticancer immunity acting through the induction of T-lymphocyte and natural killer cell proliferation. The Company previously reported positive safety and encouraging Phase 1 results with IMNN-001 administered as monotherapy or as combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and completed a Phase 1b dose-escalation trial (the OVATION 1 Study) of IMNN-001 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer.

About Epithelial Ovarian Cancer

Epithelial ovarian cancer (EOC) is the fifth deadliest malignancy among women in the United States. There are approximately 22,000 new cases of ovarian cancer every year and approximately 70% are diagnosed in advanced Stage III/IV. EOC is characterized by dissemination of tumor in the peritoneal cavity with a high risk of recurrence (75% in Stage III/IV) after surgery and chemotherapy. Since the five-year survival rates of patients with Stage III/IV disease at diagnosis are poor (41% and 20%, respectively), there remains a need for a therapy that not only reduces the recurrence rate, but also improves overall survival. The peritoneal cavity of advanced ovarian cancer patients contains the primary tumor environment and is an attractive target for a regional approach to immune modulation.

ELAHERE® Shows Overall and Progression-Free Survival Benefit Regardless of Prior PARPi Exposure or Prior Lines of Therapy in FR?-Positive Platinum-Resistant Ovarian Cancer

On September 28, 2023 ImmunoGen Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported findings from two subset analyses of the Phase 3 confirmatory MIRASOL trial (GOG 3045/ENGOT OV-55) evaluating the safety and efficacy of ELAHERE (mirvetuximab soravtansine-gynx) compared to chemotherapy in patients with folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer (PROC) (Press release, ImmunoGen, SEP 28, 2023, View Source [SID1234635499]). The findings will be presented by Dr. Toon Van Gorp in an oral presentation today at the 24th Congress of the European Society of Gynaecological Oncology (ESGO) in Istanbul, Turkey.

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"Consistent with the strong topline MIRASOL data where superiority was seen across all efficacy endpoints, these subset analyses show that the improvements in progression-free survival, objective response rates, and overall survival demonstrated in the overall study population are also observed regardless of the number of prior lines of therapy," said Toon Van Gorp, Professor of Gynaecological Oncology at the University of Leuven. "Importantly, the benefit seen with ELAHERE in patients treated with a prior PARP inhibitor is particularly encouraging, as it has been shown that PARP inhibitors have a potential negative impact on the efficacy of subsequent chemotherapies. These new data being presented at ESGO, including a consistent safety and tolerability profile, provide valuable insights for physicians into ELAHERE’s broad and meaningful benefit compared to chemotherapy and further position ELAHERE to become the new standard of care for patients with FRα-positive PROC."

MIRASOL is a randomized Phase 3 trial of ELAHERE versus investigator’s choice (IC) of single-agent chemotherapy (weekly paclitaxel, pegylated liposomal doxorubicin (PLD), or topotecan). MIRASOL enrolled 453 patients with PROC, whose tumors express high levels of FRα using what is now the Ventana FOLR1-2.1 assay, and who have been treated with one to three prior regimens. Patients were stratified by number of prior lines of therapy (14% had one prior line of therapy, 40% had two prior lines of therapy, and 46% had three prior lines of therapy) and by IC chemotherapy, with paclitaxel as the most commonly chosen (41%), followed by PLD (36%) and topotecan (23%). 62% of patients received prior bevacizumab; 55% received a prior PARP inhibitor. The primary endpoint of this trial is progression-free survival (PFS) by investigator assessment. Key secondary endpoints include objective response rate (ORR), overall survival (OS), and patient-reported outcomes (PROs).

EFFICACY OF MIRVETUXIMAB SORAVTANSINE IN FOLATE RECEPTOR ALPHA HIGH, PLATINUM-RESISTANT OVARIAN CANCER BY TYPE AND NUMBER OF PRIOR TREATMENT REGIMENS: AN EXPLORATORY ANALYSIS

Lead Author: Toon Van Gorp, MD

Date/Time: Thursday, September 28, 2023, 2:00-2:35pm TRT / 7:00-7:35am ET

Abstract: #1015 and #1056

In addition to the top-line MIRASOL data previously disclosed and subsequently presented at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, the following new data are being presented at ESGO:

ELAHERE demonstrated clinically meaningful improvement in PFS and ORR by investigator assessment and in OS compared to IC chemotherapy, regardless of prior PARPi exposure.
In patients with prior PARPi (n=251), PFS hazard ratio (HR) was 0.58 (95% CI: 0.43, 0.78; p= 0.0002); in the PARPi-naïve subset (n=191), PFS HR was 0.74 (95% CI: 0.54, 1.03, p =0.0685).
In patients with prior PARPi, ORR in the ELAHERE arm was 45% (95% CI: 36%, 54%), including 7 CRs, compared to 17% (95% CI: 11%, 25%), with no CRs, in the IC chemotherapy arm (p<0.0001); in the PARPi-naïve subset, ORR in the ELAHERE arm was 40% (95% CI: 30%, 51%), including 5 CRs, compared to 14% (95% CI: 8%, 23%), with no CRs, in the IC chemotherapy arm (p<0.0001).
In patients with prior PARPi, OS HR was 0.48 (95% CI: 0.33, 0.71; p= 0.0002); in the PARPi-naïve subset, OS HR was 0.90 (95% CI: 0.590, 1.38, p =0.6319).
ELAHERE demonstrated clinically meaningful improvements in PFS and ORR by investigator assessment and in OS compared to IC chemotherapy, regardless of prior lines of therapy (PLOT).
In patients with 1 or 2 PLOT (n=245), the PFS HR was 0.61 (95% CI: 0.45, 0.81; p=0.0007); in patients with 3 PLOT (n=208), PFS HR was 0.71 (95% CI: 0.52, 0.98; p=0.0362).
In patients with 1 or 2 PLOT, ORR in the ELAHERE arm was 46% (95% CI: 37%, 55%), including 10 complete responses (CRs), compared to 15% (95% CI: 9%, 22%), with no CRs, in the IC chemotherapy arm (p<0.0001); in patients with 3 PLOT, ORR in the ELAHERE arm was 38% (95% CI: 29%, 48%), including 2 CRs, compared to 18% (95% CI:11%, 26%), with no CRs, in the IC chemotherapy arm (p=0.0009).
In patients with 1 or 2 PLOT, OS HR was 0.66 (95% CI: 0.45, 0.98; p=0.0375); in patients with 3 PLOT, OS HR was 0.65 (95% CI: 0.43, 0.96; p=0.0308).
ELAHERE demonstrated a tolerable safety profile compared to IC chemotherapy consisting predominantly of low-grade ocular and gastrointestinal events.
In all patients, the frequency of grade 3+ treatment-emergent adverse events (TEAEs) was 42% with ELAHERE and 54% with IC chemotherapy; the frequency of serious adverse events (SAEs) was 24% with ELAHERE and 33% with IC chemotherapy; and the frequency of discontinuations due to a TEAE was 9% with ELAHERE and 16% with IC chemotherapy.
"Given ELAHERE is a potentially transformative option for those with platinum-resistant ovarian cancer, it will be important for clinicians to understand the consistency across these subset analyses from MIRASOL as they make treatment decisions with their patients," said Michael Vasconcelles, MD, ImmunoGen’s Executive Vice President, Research, Development, and Medical Affairs. "Looking forward, we are excited to submit our MAA and sBLA to the EMA and FDA, respectively, before year end while continuing to advance our broader development program as we prioritize delivering ELAHERE to eligible patients in need."

In November 2022, the US Food and Drug Administration (FDA) granted accelerated approval for ELAHERE for the treatment of adult patients with FRα-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens based on ORR and duration of response data from the pivotal SORAYA trial.

Additional information can be found at www.congress.esgo.org.

ABOUT OVARIAN CANCER

Ovarian cancer is the leading cause of death from gynecological cancers in the US. Each year, roughly 20,000 patients are diagnosed, and 13,000 patients will die. Most patients present with late-stage disease and will typically undergo surgery followed by platinum-based chemotherapy. Unfortunately, the majority of patients eventually develop platinum-resistant disease, which is difficult to treat. In this setting, standard of care single-agent chemotherapies are associated with low response rates, short durations of response, and significant toxicities.

ABOUT ELAHERE

ELAHERE (mirvetuximab soravtansine-gynx) is a first-in-class ADC comprising a folate receptor alpha-binding antibody, cleavable linker, and the maytansinoid payload DM4, a potent tubulin inhibitor designed to kill the targeted cancer cells.

Indication and Usage

ELAHERE is indicated for the treatment of adult patients with folate receptor-alpha (FRα) positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Select patients for therapy based on an FDA-approved test.

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Important Safety Information

BOXED WARNING: OCULAR TOXICITY

ELAHERE can cause severe ocular toxicities, including visual impairment, keratopathy, dry eye, photophobia, eye pain, and uveitis.
Conduct an ophthalmic exam including visual acuity and slit lamp exam prior to initiation of ELAHERE, every other cycle for the first 8 cycles, and as clinically indicated.
Administer prophylactic artificial tears and ophthalmic topical steroids.
Withhold ELAHERE for ocular toxicities until improvement and resume at the same or reduced dose.
Discontinue ELAHERE for Grade 4 ocular toxicities.
WARNINGS and PRECAUTIONS

Ocular Disorders

ELAHERE can cause severe ocular adverse reactions, including visual impairment, keratopathy (corneal disorders), dry eye, photophobia, eye pain, and uveitis.

Ocular adverse reactions occurred in 61% of patients with ovarian cancer treated with ELAHERE. Nine percent (9%) of patients experienced Grade 3 ocular adverse reactions, including visual impairment, keratopathy/keratitis (corneal disorders), dry eye, photophobia, and eye pain; and one patient (0.2%) experienced Grade 4 keratopathy. The most common (≥5%) ocular adverse reactions were visual impairment (49%), keratopathy (36%), dry eye (26%), cataract (15%), photophobia (13%), and eye pain (12%).

The median time to onset for first ocular adverse reaction was 1.2 months (range: 0.03 to 12.9). Of the patients who experienced ocular events, 49% had complete resolution and 39% had partial improvement (defined as a decrease in severity by one or more grades from the worst grade) at last follow up. Ocular adverse reactions led to permanent discontinuation of ELAHERE in 0.6% of patients.

Premedication and use of lubricating and ophthalmic topical steroids eye drops during treatment with ELAHERE are recommended. Advise patients to avoid use of contact lenses during treatment with ELAHERE unless directed by a healthcare provider.

Refer patients to an eye care professional for an ophthalmic exam including visual acuity and slit lamp exam prior to treatment initiation, every other cycle for the first 8 cycles, and as clinically indicated. Promptly refer patients to an eye care professional for any new or worsening ocular signs and symptoms.

Monitor for ocular toxicity and withhold, reduce, or permanently discontinue ELAHERE based on severity and persistence of ocular adverse reactions.

Pneumonitis

Severe, life-threatening, or fatal interstitial lung disease, including pneumonitis, can occur in patients treated with ELAHERE. Pneumonitis occurred in 10% of patients treated with ELAHERE, including 0.8% with Grade 3 events, and 1 patient (0.2%) with a Grade 4 event. One patient (0.2%) died due to respiratory failure in the setting of pneumonitis and lung metastases.

Monitor patients for pulmonary signs and symptoms of pneumonitis. Infectious, neoplastic, and other causes for symptoms should be excluded through appropriate investigations.

Withhold ELAHERE for patients who develop persistent or recurrent Grade 2 pneumonitis until symptoms resolve to ≤ Grade 1 and consider dose reduction. Permanently discontinue ELAHERE in all patients with Grade 3 or 4 pneumonitis. Patients who are asymptomatic may continue dosing of ELAHERE with close monitoring.

Peripheral Neuropathy (PN)

PN occurred in 36% of patients with ovarian cancer treated with ELAHERE across clinical trials; 2% of patients experienced Grade 3 PN. PN adverse reactions included peripheral neuropathy (19%), peripheral sensory neuropathy (9%), paraesthesia (6%), neurotoxicity (3%), hypoaesthesia (2%), peripheral motor neuropathy (1%), neuralgia (0.4%), polyneuropathy (0.2%) and oral hypoesthesia (0.2%).

Monitor patients for signs and symptoms of neuropathy. For patients experiencing new or worsening PN, withhold dosage, dose reduce, or permanently discontinue ELAHERE based on the severity of PN.

Embryo-Fetal Toxicity

Based on its mechanism of action, ELAHERE can cause embryo-fetal harm when administered to a pregnant woman because it contains a genotoxic compound (DM4) and affects actively dividing cells.

Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with ELAHERE and for 7 months after the last dose.

ADVERSE REACTIONS

Serious adverse reactions occurred in 31% of patients. The most common (≥2%) serious adverse reactions were intestinal obstruction (8%), ascites (4%), infection (3%), and pleural effusion (3%). Fatal adverse reactions occurred in 2% of patients, including small intestinal obstruction (1%) and pneumonitis (1%).

Permanent discontinuation of ELAHERE due to adverse reactions occurred in 11% of patients. The most common (≥2%) adverse reactions leading to permanent discontinuation were intestinal obstruction (2%) and thrombocytopenia (2%). One patient (0.9%) permanently discontinued ELAHERE due to visual impairment (unilateral decrease to BCVA < 20/200 that resolved to baseline after discontinuation).

Dosage delays of ELAHERE due to an adverse reaction occurred in 39% of patients. Adverse reactions which required dosage delays in ≥3% of patients included visual impairment (15%), keratopathy (11%), neutropenia (6%), dry eye (5%), cataracts (3%) and increased gamma-glutamyltransferase (3%).

Dose reductions of ELAHERE due to an adverse reaction occurred in 20% of patients. Adverse reactions which required dose reductions in ≥3% of patients included visual impairment (9%) and keratopathy (7%).

The most common (≥20%) adverse reactions, including laboratory abnormalities, were vision impairment, fatigue, increased aspartate aminotransferase, nausea, increased alanine aminotransferase, keratopathy, abdominal pain, decreased lymphocytes, peripheral neuropathy, diarrhea, decreased albumin, constipation, increased alkaline phosphatase, dry eye, decreased magnesium, decreased leukocytes, decreased neutrophils, and decreased hemoglobin.

DRUG INTERACTIONS

Strong CYP3A4 Inhibitors

DM4 is a CYP3A4 substrate. Concomitant use of ELAHERE with strong CYP3A4 inhibitors may increase unconjugated DM4 exposure, which may increase the risk of ELAHERE adverse reactions. Closely monitor patients for adverse reactions with ELAHERE when used concomitantly with strong CYP3A4 inhibitors.

USE IN SPECIAL POPULATIONS

Lactation

Advise women not to breastfeed during treatment with ELAHERE and for at least 1 month after the last dose.

Pediatric Use

Safety and effectiveness of ELAHERE have not been established in pediatric patients.

Hepatic Impairment

Avoid use of ELAHERE in patients with moderate or severe hepatic impairment (total bilirubin >1.5 ULN).

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

BioLineRx Announces Encouraging Data from Pilot Phase of Phase 2 Combination Clinical Trial with Motixafortide in First-Line Pancreatic Cancer (PDAC)

On September 28, 2023 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, reported encouraging data from the single-arm pilot phase of the investigator-initiated CheMo4METPANC Phase 2 combination clinical trial evaluating the company’s CXCR4 inhibitor motixafortide, the PD-1 inhibitor cemiplimab, and standard of care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel alone, in first-line pancreatic cancer (PDAC) (Press release, BioLineRx, SEP 28, 2023, View Source [SID1234635498]).

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The data were published in an online abstract as part of the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Pancreatic Cancer taking place in Boston, Massachusetts from September 27-30, 2023. An oral presentation of the data will take place later today, September 28, 2023.

The pilot phase of the Phase 2 study enrolled 11 patients with metastatic pancreatic cancer. As of May 2023, 6 patients (55%) experienced a partial response (PR) of which 4 (36%) were confirmed PRs with one patient experiencing resolution of the hepatic (liver) metastatic lesion. Three patients (27%) experienced stable disease, resulting in a disease control rate of 82%. These findings compare favorably to historic partial response and disease control rates of 23% and 48%, respectively, reported with the current standard of care, the chemotherapy combination gemcitabine and nab-paclitaxel.

"These initial data from the pilot phase of this ongoing Phase 2 study give us hope that motixafortide could potentially serve as the backbone of a new treatment regimen for PDAC, which is among the most difficult cancers to treat," said Philip Serlin, Chief Executive Officer of BioLineRx Ltd. "We are deeply committed to this important collaboration with Columbia University investigators and eagerly look forward to the data from the randomized phase of the trial."

Based on these pilot data, earlier this year, the CheMo4METPANC Phase 2 trial was amended to become a randomized study, with planned enrollment increasing from 30 to 102 patients. The trial, sponsored by Columbia University, is the first large, multi-center, randomized study evaluating motixafortide with a PD-1 inhibitor and first-line PDAC chemotherapies. A poster of the amended clinical trial design was presented at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, held June 2-6 in Chicago, Illinois (see abstract).

Pancreatic ductal adenocarcinoma (PDAC) is the most common type of pancreatic cancer and is expected to be the second leading cause of cancer-related death in the U.S. by 2023. Because it is typically diagnosed at later stages, greater than 80 percent of pancreatic cancer is inoperable. Most pancreatic cancer is incurable and unfortunately newer immunotherapy approaches, while beneficial against other solid tumor types, have had limited efficacy in pancreatic cancer due to immunosuppressive pathways.

An earlier single-arm, Phase 2a clinical trial (COMBAT/KEYNOTE-202) and pre-clinical studies evaluating motixafortide in combination with PD-1 immunotherapies and chemotherapies in PDAC have been promising, suggesting the ability of motixafortide to support an immune response.

Presentation at AACR (Free AACR Whitepaper) Special Conference in Cancer Research: Pancreatic Cancer

Westin Copley Place, Boston Massachusetts

Plenary Session Details

Title: CheMo4METPANC: Combination Chemotherapy (gemcitabine and nab-paclitaxel), chemokine (C-X-C) Motif receptor 4 inhibitor (motixafortide), and immune checkpoint blockade (cemiplimab) in METastatic treatment-naïve PANCreatic adenocarcinoma

Presenter: Gulam A. Manji, MD, PhD, Columbia University Irving Medical Center/New York Presbyterian, New York, N.Y.

Session: Plenary Session 3: Clinical Updates

Date: Thursday, September 28, 2023

Time: 2:30-4:40 pm EDT

About CheMo4METPANC Phase 2 Clinical Trial

The multi-center CheMo4METPANC Phase 2 clinical trial is a randomized, investigator-initiated clinical trial in first line metastatic pancreatic cancer. Sponsored by Columbia University, the study is evaluating the combination of CXCR4 inhibitor motixafortide, PD-1 inhibitor cemiplimab, and standard of care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel, alone in 102 patients. The trial’s primary endpoint is progression free survival (PFS). Secondary objectives include safety, response rate, disease control rate, duration of clinical benefit and overall survival.

About Pancreatic Cancer

Pancreatic cancer has a low rate of early diagnosis and a poor prognosis. In the United States in 2023, an estimated 64,000 adults will be diagnosed with the disease, which accounts for approximately 3% of all cancers in the U.S. and about 7% of all cancer deaths. Worldwide, an estimated 496,000 people were diagnosed with the disease in 2020. In the U.S., if the cancer is detected at an early stage when surgical removal of the tumor is possible, the 5-year relative survival rate is 44%. About 12% of people are initially diagnosed at this stage. If the cancer has spread to surrounding tissues or organs, the 5-year relative survival rate is 15%. For the 52% of patients who are initially diagnosed with metastatic cancer, the 5-year relative survival rate is 3%.[i] In particular, hepatic (liver) metastases are a critical risk factor driving poor prognoses for patients with metastatic PDAC. These data highlight the need for the development of new therapeutic options.

About Motixafortide in Cancer Immunotherapy

Motixafortide inhibits CXCR4, a chemokine receptor and a well validated therapeutic target that is over-expressed in many human cancers including pancreatic ductal adenocarcinoma (PDAC). Motixafortide leverages the expression of the CXCR4 receptor on different immune cells and potentiates the immune system against the tumor. Among CXCR4-expressing immune cells, some exhibit anti-tumoral activity, such as effector T cells and some exhibit pro-tumoral activity and support tumor growth. By blocking the CXCR4 receptor, motixafortide was shown in a Phase 2 study in pancreatic cancer patients to enhance anti-tumoral activity and to ameliorate the pro-tumoral activities by modulating the effector/suppressor cell ratio towards a proinflammatory profile.

Ariceum Therapeutics’ targeted radiopharmaceutical 177Lu-satoreotide exhibits promising clinical response and good tolerability profile in patients with advanced neuroendocrine tumours

On September 28, 2023 Ariceum Therapeutics (Ariceum), a private biotech company developing radiopharmaceutical products for the diagnosis and treatment of certain hard-to-treat cancers, reported the publication of positive results from a Phase I/II trial of its radiopharmaceutical 177Lu-satoreotide tetraxetan(satoreotide) in patients with previously treated, progressive neuroendocrine tumours (NETs), in the European Journal of Nuclear Medicine and Molecular Imaging (Press release, Ariceum Therapeutics, SEP 28, 2023, View Source [SID1234635497]).

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Satoreotide combines Ariceum’s proprietary peptide satoreotide – a first-in-class and best-in-class antagonist of the somatostatin receptor 2 (SSTR2) – with the radioactive isotope ‘payload’ 177Lutetium. SSTR2 is a cell surface protein often overexpressed in certain cancers, including NETs and small cell lung cancer (SCLC).

The Phase I/II trial was initiated by Ipsen, and recently completed after Ariceum acquired satoreotide from Ipsen. This international study was conducted in 7 countries – Australia, Austria, Canada, Denmark, France, Switzerland, and the UK – and enrolled 40 patients with advanced, SSTR2-positive NETs. The primary tumours of the patients included progressive, grade 1 and 2 (≈60%) gastroenteropancreatic (GEP), and (a)typical lung NETs, paraganglioma, and pheochromocytoma. All patients had undergone several lines of treatment, including chemo- and/or radiotherapy (45%), before they were treated with 177Lu-satoreotide. Most patients received three infusions of satoreotide, with the median cumulative radiation dose being 13.0 GBq.

Of the 38 patients for whom full results were obtained, 28 (73.7%) achieved stable disease, as determined eight weeks after the last infusion. A further 8 (21.1%) experienced a partial response (a reduction in tumour size) – giving a total Disease Control Rate (DCR) of 94.7%. 17 of the 40 patients (42.5%) experienced grade ≥3 treatment‑related adverse events, the most common being lymphopenia, thrombocytopenia, and neutropenia. Two patients developed myeloid neoplasms considered treatment-related by the investigator.

The authors of the study, titled "A phase I/II study of the safety and efficacy of [177Lu]Lu‑satoreotide tetraxetan in advanced somatostatin receptor‑positive neuroendocrine tumours", concluded satoreotide "has an acceptable safety profile with a promising clinical response in patients with progressive, SSTR-positive NETs". They also discussed that the lower administered activity, 3 cycles of 4.5 GBq compared to 4 cycles of 7.4 GBq with 177Lu-DOTATATE, may offer advantages regarding the treatment burden for patients, but also in terms of reduction of nuclear waste and direct radioisotope costs. A 5-year follow-up study is ongoing.

Manfred Rüdiger, PhD, Chief Executive Officer of Ariceum Therapeutics, said: "These exciting new data demonstrate the great potential of our targeted radiopharmaceutical, satoreotide, for treating patients with advanced neuroendocrine tumours. Not only did a high proportion of treated patients achieve stable disease or better, but they did so on a lower dose of radiation than the investigators initially thought was needed. These results will greatly assist Ariceum in further developing satoreotide for hard-to-treat neuroendocrine cancers such as small cell lung cancer."

Further details on the study can be found on Clinical Trials, under identifier NCT05017662.

Citation:

Wild, D., Grønbæk, H., Navalkissoor, S. et al. A phase I/II study of the safety and efficacy of [177Lu]Lu-satoreotide tetraxetan in advanced somatostatin receptor-positive neuroendocrine tumours. Eur J Nucl Med Mol Imaging (2023). View Source