New Phase 1 Dose Escalation Cohorts Data for Aulos Bioscience’s AU-007 Show Favorable Pharmacodynamic Effects at 2023 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics

On October 14, 2023 Aulos Bioscience, an immuno-oncology company working to revolutionize cancer care through the development of potentially best-in-class IL-2 therapeutics, reported new pharmacodynamic data from the ongoing Phase 1 dose escalation portion of its Phase 1/2 clinical trial of AU-007, the first human monoclonal antibody designed using artificial intelligence to be tested in a clinical trial (Press release, Aulos Bioscience, OCT 14, 2023, View Source [SID1234635968]). The data were shared in a poster presentation at the 2023 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in Boston, Massachusetts.

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The study evaluated AU-007 as a monotherapy treatment or in a combination therapy regimen with low-dose subcutaneous Proleukin (aldesleukin; recombinant human IL-2). The data demonstrate that AU-007 redirects interleukin-2 (IL-2) from regulatory T cells (Tregs) toward effector T cells (Teffs) and natural killer (NK) cells, which can kill tumor cells. The data also show a reduction in eosinophils, which at high cell counts can be associated with toxicity in the lungs.

"We believe AU-007 may be therapeutically effective in solid tumor cancers, and the cell types of interest in this study are trending in the right directions," said Aron Knickerbocker, Aulos Bioscience’s chief executive officer. "These data continue to demonstrate favorable and unique pharmacodynamic effects in the IL-2 class. When AU-007 is administered to patients as monotherapy or in combination with low doses of Proleukin, the data show a decrease in immunosuppressive Tregs and increases in the numbers of Teffs and NK cells, both of which are cell types capable of killing tumor cells. To our knowledge, no other IL-2 therapy has shown such a profile of immune cell changes and the resulting change in the ratio of effector to suppressor immune cells. We also observed immune activation as assessed by interferon-gamma levels, which tend to increase following AU-007 with Proleukin. We will share the results of further pharmacodynamic investigations of AU-007 in combination with higher doses of Proleukin in the future. We also look forward to presenting new clinical efficacy and safety data from the Phase 1 dose escalation cohorts later this year."

Created by Biolojic Design, AU-007 is a human IgG1 monoclonal antibody designed using artificial intelligence to harness the power of IL-2 to eradicate solid tumors. The antibody’s novel mechanism of action lies in its ability to bind precisely to IL-2 instead of IL-2 receptors. By binding only to the portion of IL-2 that binds to CD25, AU-007 prevents IL-2 from binding to high-affinity IL-2 receptors on Tregs and eosinophils and redirects IL-2 to medium-affinity receptors on NK cells and effector T cells. This allows effector T cells and NK cells to expand and kill tumor cells.

The Phase 1 dose escalation study presented at the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference shows that AU-007 redirected IL-2 from Tregs toward effector T cells and NK cells and expanded those cells with and without low doses of Proleukin, which is consistent with the antibody’s mechanism of action. This overall effect increased with time on Proleukin given every two weeks with AU-007, or with higher dose levels of Proleukin. These changes were observed across a broad range of cancer types and were not associated with any drug-related events. Additionally, AU-007 treatment led to decreases in eosinophils and increases in interferon-gamma with and without Proleukin, which supports the antibody’s proposed activity.

The Phase 1/2 clinical trial of AU-007 is a two-part, open label, first-in-human study evaluating the safety, tolerability, immunogenicity and clinical activity of AU-007 in patients with unresectable locally advanced or metastatic cancer. The trial is currently enrolling patients at multiple site locations in the United States and Australia. The company anticipates transitioning to the Phase 2 portion of the AU-007 study by year-end.

The poster presentation is available on the Aulos Bioscience website in the Abstracts and Publications section.

About AU-007

AU-007 is a computationally designed, human IgG1 monoclonal antibody that is highly selective to the CD25-binding portion of IL-2. With a mechanism of action unlike any other IL-2 therapeutic in development, AU-007 leverages IL-2 to reinforce anti-tumor immune effects. This is achieved by preventing IL-2, either exogenous or secreted by effector T cells, from binding to trimeric receptors on regulatory T cells while still allowing IL-2 to bind and expand effector T cells and NK cells. This prevents the negative feedback loop caused by other IL-2-based treatments and biases the immune system toward activation over suppression. AU-007 also prevents IL-2 from binding to CD25-containing receptors on vasculature and pulmonary endothelium, which may significantly reduce the vascular leak syndrome and pulmonary edema associated with high-dose IL-2 therapy.

U.S. Food and Drug Administration Approves Opdivo® (nivolumab) as Adjuvant Treatment for Eligible Patients with Completely Resected Stage IIB or Stage IIC Melanoma

On October 13, 2023 Bristol Myers Squibb (NYSE: BMY) reported that Opdivo(nivolumab) was approved by the U.S. Food and Drug Administration (FDA) for the adjuvant treatment of adult and pediatric patients 12 years and older with completely resected stage IIB or IIC melanoma, expanding upon the existing adjuvant indication for Opdivo and further reinforcing the company’s legacy of providing treatment options for melanoma patients (Press release, Bristol-Myers Squibb, OCT 13, 2023, View Source [SID1234635945]). The approval is based on the Phase 3 CheckMate -76K trial, which compared Opdivo (n=526) to placebo (n=264).1,2

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In the trial, Opdivo reduced the risk of recurrence, new primary melanoma, or death in patients with completely resected stage IIB or IIC melanoma by 58% compared to placebo (Hazard Ratio [HR] 0.42; 95% Confidence Interval [CI]: 0.30-0.59; P<0.0001).1 At one year, the recurrence-free survival (RFS) rate was 89% (95% CI: 86-92) for Opdivo versus 79% (95% CI: 74-84) for placebo.3 Additionally, in a pre-specified, exploratory subgroup analysis, the RFS unstratified HR was 0.34 (95% CI: 0.20-0.56) in patients with stage IIB melanoma, and 0.51 (95% CI: 0.32-0.81) in stage IIC melanoma patients.3 One-year RFS rates by stage for patients who received Opdivo were 93% (95% CI: 89–95) in stage IIB versus 84% (95% CI: 77–89) with placebo, and 84% (95% CI: 78–88) in stage IIC versus 72% (95% CI: 62–80) with placebo.3

"Following surgical removal of melanoma, patients may believe they are free of disease," said John M. Kirkwood, M.D., Distinguished Professor of Medicine at the University of Pittsburgh School of Medicine and Co-Director of the Melanoma Center at UPMC Hillman Cancer Center. "However, within five years of diagnosis, one-third of patients with surgically resected stage IIB and nearly one-half of patients with surgically resected IIC melanoma see their cancer return, underscoring the need for additional treatment options that may help reduce the risk of cancer coming back.4 The significant recurrence-free survival improvement observed with nivolumab in CheckMate -76K is an important step forward for these patients."1

Opdivo is associated with the following Warnings & Precautions: severe and fatal immune-mediated adverse reactions, including pneumonitis, colitis, hepatitis and hepatotoxicity, endocrinopathies, dermatologic adverse reactions, nephritis with renal dysfunction, other immune-mediated adverse reactions; infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation (HSCT); embryo-fetal toxicity; and increased mortality in patients with multiple myeloma when Opdivo is added to a thalidomide analogue and dexamethasone, which is not recommended outside of controlled clinical trials.1 Please see Important Safety Information below.

"Stage IIB and IIC melanoma patients may still face the threat of disease recurrence, despite the benefit of surgery, which can impact outcomes," said Catherine Owen, senior vice president and general manager, U.S. Cardiovascular, Immunology and Oncology at Bristol Myers Squibb.4,5 "This approval builds on our existing adjuvant indication in completely resected stage III or IV disease and now provides eligible patients with completely resected stage IIB or IIC melanoma an additional treatment option which may help prevent recurrence. BMS remains committed to its goal of helping improve patient outcomes in melanoma and bringing immunotherapy to more patients, including in the earlier stages of disease."1

The FDA previously approved Opdivo for the adjuvant treatment of adult and pediatric patients 12 years and older with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection, based upon data from the CheckMate -238 trial.1

Additional CheckMate -76K follow-up data will be presented at the Society for Melanoma Research Annual Meeting in November.

About CheckMate -76K

CheckMate -76K is a Phase 3, randomized, double-blind study evaluating adjuvant Opdivo (nivolumab) 480 mg IV Q4W (n=526) versus placebo IV Q4W (n=264) in patients with completely resected stage IIB or IIC melanoma.1,2

The primary endpoint of the trial is recurrence-free survival (RFS) as assessed by the investigator.1 Secondary endpoints of the trial include overall survival (OS), distant metastasis-free survival (DMFS), progression-free survival through next-line therapy (PFS2), and safety.3 Patients were treated for up to 1 year, or until disease recurrence or unacceptable toxicity.1 The trial excluded patients with ocular/uveal or mucosal melanoma, autoimmune disease, any condition requiring systemic treatment with either corticosteroids (≥10 mg daily prednisone or equivalent) or other immunosuppressive medications, as well as patients with prior therapy for melanoma except surgery.1

The FDA-approved dosing for Opdivo for adjuvant treatment of adult and pediatric patients 12 years and older with completely resected stage IIB/C melanoma and weighing ≥40kg is Opdivo 240 mg every 2 weeks or 480 mg every 4 weeks administered as an IV infusion over 30 minutes until disease recurrence or unacceptable toxicity for up to 1 year.1 The FDA-approved dosing for Opdivo in pediatric patients age 12 years and older and weighing <40kg is Opdivo 3 mg/kg every 2 weeks or 6 mg/kg every 4 weeks administered as an IV infusion over 30 minutes until disease recurrence or unacceptable toxicity for up to 1 year.1

About Melanoma

Melanoma is a form of skin cancer characterized by the uncontrolled growth of pigment-producing cells (melanocytes) located in the skin.6 In the United States, approximately 97,610 new diagnoses of melanoma and about 7,990 related deaths are estimated for 2023.7 Stage II makes up approximately 16.5% of newly diagnosed stage I and II melanomas, and approximately half of these stage II diagnoses are IIB and IIC.4 Surgery (resection) remains a standard of care for stage IIB or IIC melanoma, but approximately one-third of patients with surgically resected stage IIB and nearly one-half of patients with surgically resected stage IIC melanoma experience recurrence within five years after diagnosis.4,8 As melanoma progresses to more advanced disease, it becomes more challenging to treat and survival rates decline.6

INDICATION

OPDIVO is indicated for the adjuvant treatment of adult and pediatric patients 12 years and older with completely resected Stage IIB, Stage IIC, Stage III, or Stage IV melanoma.

IMPORTANT SAFETY INFORMATION

Severe and Fatal Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions listed herein may not include all possible severe and fatal immune-mediated adverse reactions.

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue. While immune-mediated adverse reactions usually manifest during treatment, they can also occur after discontinuation of OPDIVO. Early identification and management are essential to ensure safe use of OPDIVO. Monitor for signs and symptoms that may be clinical manifestations of underlying immune-mediated adverse reactions. Evaluate clinical chemistries including liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment with OPDIVO. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue OPDIVO depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). In general, if OPDIVO interruption or discontinuation is required, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reactions are not controlled with corticosteroid therapy. Toxicity management guidelines for adverse reactions that do not necessarily require systemic steroids (e.g., endocrinopathies and dermatologic reactions) are discussed below.

Immune-Mediated Pneumonitis

OPDIVO can cause immune-mediated pneumonitis. The incidence of pneumonitis is higher in patients who have received prior thoracic radiation. In patients receiving OPDIVO monotherapy, immune-mediated pneumonitis occurred in 3.1% (61/1994) of patients, including Grade 4 (<0.1%), Grade 3 (0.9%), and Grade 2 (2.1%).

Immune-Mediated Colitis

OPDIVO can cause immune-mediated colitis. A common symptom included in the definition of colitis was diarrhea. Cytomegalovirus (CMV) infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. In patients receiving OPDIVO monotherapy, immune-mediated colitis occurred in 2.9% (58/1994) of patients, including Grade 3 (1.7%) and Grade 2 (1%).

Immune-Mediated Hepatitis and Hepatotoxicity

OPDIVO can cause immune-mediated hepatitis. In patients receiving OPDIVO monotherapy, immune-mediated hepatitis occurred in 1.8% (35/1994) of patients, including Grade 4 (0.2%), Grade 3 (1.3%), and Grade 2 (0.4%).

Immune-Mediated Endocrinopathies

OPDIVO can cause primary or secondary adrenal insufficiency, immune-mediated hypophysitis, immune-mediated thyroid disorders, and Type 1 diabetes mellitus, which can present with diabetic ketoacidosis. Withhold OPDIVO depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment, including hormone replacement as clinically indicated. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism; initiate hormone replacement as clinically indicated. Thyroiditis can present with or without endocrinopathy.

Hypothyroidism can follow hyperthyroidism; initiate hormone replacement or medical management as clinically indicated. Monitor patients for hyperglycemia or other signs and symptoms of diabetes; initiate treatment with insulin as clinically indicated.

In patients receiving OPDIVO monotherapy, adrenal insufficiency occurred in 1% (20/1994), including Grade 3 (0.4%) and Grade 2 (0.6%).

In patients receiving OPDIVO monotherapy, hypophysitis occurred in 0.6% (12/1994) of patients, including Grade 3 (0.2%) and Grade 2 (0.3%).

In patients receiving OPDIVO monotherapy, thyroiditis occurred in 0.6% (12/1994) of patients, including Grade 2 (0.2%).

In patients receiving OPDIVO monotherapy, hyperthyroidism occurred in 2.7% (54/1994) of patients, including Grade 3 (<0.1%) and Grade 2 (1.2%).

In patients receiving OPDIVO monotherapy, hypothyroidism occurred in 8% (163/1994) of patients, including Grade 3 (0.2%) and Grade 2 (4.8%).

In patients receiving OPDIVO monotherapy, diabetes occurred in 0.9% (17/1994) of patients, including Grade 3 (0.4%) and Grade 2 (0.3%), and 2 cases of diabetic ketoacidosis.

Immune-Mediated Nephritis with Renal Dysfunction

OPDIVO can cause immune-mediated nephritis. In patients receiving OPDIVO monotherapy, immune-mediated nephritis and renal dysfunction occurred in 1.2% (23/1994) of patients, including Grade 4 (<0.1%), Grade 3 (0.5%), and Grade 2 (0.6%).

Immune-Mediated Dermatologic Adverse Reactions

OPDIVO can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug rash with eosinophilia and systemic symptoms (DRESS) has occurred with PD-1/PD-L1 blocking antibodies. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate nonexfoliative rashes.

Withhold or permanently discontinue OPDIVO depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information).

In patients receiving OPDIVO monotherapy, immune-mediated rash occurred in 9% (171/1994) of patients, including Grade 3 (1.1%) and Grade 2 (2.2%).

Other Immune-Mediated Adverse Reactions

The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received OPDIVO monotherapy or were reported with the use of other PD-1/PD-L1 blocking antibodies. Severe or fatal cases have been reported for some of these adverse reactions: cardiac/vascular: myocarditis, pericarditis, vasculitis; nervous system: meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barré syndrome, nerve paresis, autoimmune neuropathy; ocular: uveitis, iritis, and other ocular inflammatory toxicities can occur; gastrointestinal: pancreatitis to include increases in serum amylase and lipase levels, gastritis, duodenitis; musculoskeletal and connective tissue: myositis/polymyositis, rhabdomyolysis, and associated sequelae including renal failure, arthritis, polymyalgia rheumatica; endocrine: hypoparathyroidism; other (hematologic/immune): hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis (HLH), systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection.

Some ocular IMAR cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada–like syndrome, which has been observed in patients receiving OPDIVO, as this may require treatment with systemic corticosteroids to reduce the risk of permanent vision loss.

Infusion-Related Reactions

OPDIVO can cause severe infusion-related reactions. Discontinue OPDIVO in patients with severe (Grade 3) or life-threatening (Grade 4) infusion-related reactions. Interrupt or slow the rate of infusion in patients with mild (Grade 1) or moderate (Grade 2) infusion-related reactions. In patients receiving OPDIVO monotherapy as a 60-minute infusion, infusion-related reactions occurred in 6.4% (127/1994) of patients. In a separate trial in which patients received OPDIVO monotherapy as a 60-minute infusion or a 30-minute infusion, infusion-related reactions occurred in 2.2% (8/368) and 2.7% (10/369) of patients, respectively. Additionally, 0.5% (2/368) and 1.4% (5/369) of patients, respectively, experienced adverse reactions within 48 hours of infusion that led to dose delay, permanent discontinuation or withholding of OPDIVO.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation

Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with OPDIVO. Transplant-related complications include hyperacute graft-versus-host-disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease (VOD) after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between OPDIVO and allogeneic HSCT.

Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risks of treatment with OPDIVO prior to or after an allogeneic HSCT.

Embryo-Fetal Toxicity

Based on its mechanism of action and findings from animal studies, OPDIVO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with OPDIVO and for at least 5 months after the last dose.

Increased Mortality in Patients with Multiple Myeloma when OPDIVO is Added to a Thalidomide Analogue and Dexamethasone

In randomized clinical trials in patients with multiple myeloma, the addition of OPDIVO to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with multiple myeloma with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials.

Lactation

There are no data on the presence of OPDIVO in human milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in breastfed children, advise women not to breastfeed during treatment and for 5 months after the last dose.

Serious Adverse Reactions

In Checkmate 76K, serious adverse reactions occurred in 18% of patients receiving OPDIVO (n=524). Adverse reactions which resulted in permanent discontinuation of OPDIVO in >1% of patients included arthralgia (1.7%), rash (1.7%), and diarrhea (1.1%). A fatal adverse reaction occurred in 1 (0.2%) patient (heart failure and acute kidney injury). The most frequent Grade 3-4 lab abnormalities reported in ≥1% of OPDIVO-treated patients were increased lipase (2.9%), increased AST (2.2%), increased ALT (2.1%), lymphopenia (1.1%), and decreased potassium (1.0%).

Common Adverse Reactions

In Checkmate -76K, the most common adverse reactions (≥20%) reported with OPDIVO (n=524) were fatigue (36%), musculoskeletal pain (30%), rash (28%) diarrhea (23%) and pruritis (20%).

Please see US Full Prescribing Information for OPDIVO.

Scorpion Therapeutics Presents Preclinical Data for STX-241, a Fourth-Generation EGFR Inhibitor, at the AACR-NCI-EORTC Symposium 2023

On October 13, 2023 Scorpion Therapeutics, Inc. ("Scorpion"), a pioneering clinical-stage oncology company redefining the frontier of precision medicine through its Precision Oncology 2.0 strategy, reported preclinical data for STX-241, an orally bioavailable, highly selective, central nervous system ("CNS")-penetrant and potentially best-in-class fourth generation epidermal growth factor receptor ("EGFR") tyrosine kinase inhibitor ("TKI") designed to inhibit C797S mutations with a co-occurring EGFR exon 19 deletion or exon 21 mutation ("double mutant") in non-small cell lung cancer ("NSCLC") (Press release, Scorpion Therapeutics, OCT 13, 2023, View Source [SID1234635964]). Discovered by Scorpion, STX-241 is currently being developed in collaboration with Pierre Fabre Laboratories, a leading French medical and beauty care company with 4 decades of experience in innovation, development, manufacturing, and commercialization in oncology. The data will be presented today in a poster session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper), the National Cancer Institute, and the European Organisation for Research and Treatment of Cancer ("AACR-NCI-EORTC") Symposium 2023 in Boston, Massachusetts.

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"We are pleased to share the first preclinical data for STX-241, the second highly selective development candidate from our franchise of next-generation EGFR inhibitors for the treatment of NSCLC that we are developing with our partner, Pierre Fabre Laboratories," said Axel Hoos, M.D., Ph.D., Chief Executive Officer of Scorpion. "These data bolster our belief that STX-241 can overcome the challenges of currently approved EGFR-targeting medicines that are limited in their impact by the emergence of acquired resistance mutations. We look forward to building on these encouraging findings and submitting an investigational new drug ("IND") application in the first half of next year, as we work to deliver better outcomes for people with ‘double mutant’ NSCLC."

NSCLC is the most common form of lung cancer and EGFR mutations are one of its most common disease drivers, occurring in up to 38 percent of tumors1,2,3. The current standard-of-care therapy for NSCLC patients with EGFR exon 19 or 21 mutations is osimertinib; however, co-occurring L858R/C797x or ex19del/C797x mutations ("double mutants") are emerging as an on-target resistance mechanism in a subset of these patients, with the most recent data analyses suggesting mutation frequencies of approximately 12.5%4,5,6,7,8. There are currently no approved therapeutic options for patients that develop "double mutant" EGFR NSCLC. In addition, the relative lack of CNS penetrance with first-generation inhibitors may limit durable clinical responses to treatment, further underscoring the need for new therapies, particularly in patients with CNS metastases.

"The strong pre-clinical profile of STX-241 is a primary reason we believe in its potential to play a meaningful role in treating patients with NSCLC who have limited options," said Francesco Hofmann, Head of Research and Development for Medical Care at Pierre Fabre Laboratories. "We look forward to working with Scorpion to advance it into the clinic."

In the poster presented at AACR (Free AACR Whitepaper)-NCI-EORTC, Scorpion scientists will share data from preclinical studies characterizing the potency and selectivity of STX-241, as well as its ability to penetrate the CNS. In these studies, STX-241 demonstrated strong biochemical inhibition of EGFR double mutant kinase activity, as well as strong C797S double mutant potency and selectivity across both proliferation and target engagement assays, exceeding clinical-stage competitor benchmarks in each of these model systems.

In addition, STX-241 demonstrated strong in vivo antitumor activity across a variety of single and double-mutant cell line-derived xenograft ("CDX") models, effectively retaining activity in the presence of the C797 mutation, and CNS penetrance on-par with osimertinib, supporting its potential to address CNS metastases.

"We designed STX-241 as a potentially best-in-class small molecule for NSCLC patients who progress following first-line therapy," said Darrin Stuart, Ph.D., Chief Scientific Officer of Scorpion Therapeutics. "Together, the data presented at AACR (Free AACR Whitepaper)-NCI-EORTC underscore STX-241’s potential to provide a better option for patients who develop ‘double mutant’ disease, for which there is currently no approved treatment, as well as address the CNS metastases that occur in patients who progress on osimertinib."

STX-241 is the third development candidate nominated by Scorpion in less than three years, and the second highly selective EGFR development candidate from the Company’s franchise of next-generation mutant EGFR inhibitors for the treatment of NSCLC. Scorpion is also developing STX-721, a potentially best-in-class exon 20 mutant EGFR inhibitor, which entered the clinic earlier this month.

The poster can be viewed in the Publications & Presentations section of Scorpion’s website: View Source

About STX-241

STX-241 is a fourth generation, orally delivered, CNS-penetrant small molecule designed with potentially best-in-class selectivity to target resistance mutations at C797S. Scorpion estimates that up to 3,000 patients per year in the United States, or up to 12.5 percent of NSCLC patients with Exon 19 or 21 mutations, develop resistance mutations at C797S. STX-241 is currently advancing through preclinical studies, and Scorpion expects to submit an IND to the U.S. FDA in the first half of 2024.

ABION Presents New Efficacy Data for ABN401, a Novel MET TKI for Advanced Non-Small Cell Lung Cancer

On October 13, 2023 ABION (KOSDAQ: 203400) reported data from an ongoing Phase 2 trial of ABN401, the company’s wholly owned tyrosine kinase inhibitor intended to treat adult patients with advanced non-small cell lung cancer that exhibit the MET Exon 14 skipping mutation (Press release, ABION, OCT 13, 2023, View Source [SID1234635963]). In eight treatment-naïve patients, the objective response rate was 75% (6/8), and in the evaluable population of 17 patients, the objective response rate was 52.9% (9/17).

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Patients were dosed 800 mg QD and the median duration of response was 5.4 months as of the data cut-off on July 25, 2023. The data are not mature enough to measure progression-free survival and duration of response. All patients are MET inhibitor naïve. Overall efficacy was comparable to currently marketed drugs and demonstrate superior efficacy in treatment-naïve patients.

The cut-off data reaffirm ABN401’s excellent safety profile as shown in an earlier Phase 1 trial. A total of 24 patients received ABN401 800 mg QD, including 17 METex14 NSCLC patients. Of 24 patients evaluable for safety, 2 (8.3%) experienced grades ≥3 treatment-related adverse events (TRAE). The most common treatment-related adverse events were nausea (70.8%), vomiting (29.2%), and diarrhea (33.3%). Grade ≥3 treatment-emergent adverse events occurred in 20.8% (5/24) patients. Treatment-related adverse events leading to permanent discontinuation occurred in none of the patients (0/24). Peripheral edema was observed in 29.2% (grade 1-2) and there was no grade 3 or greater event in all safety-evaluable populations, and 17.6% (grade 1-2) and 0% (grade 3) in METex14 NSCLC population. None of the patients discontinued treatment due to SAE or TRAE.

For the 17 METex14 NSCLC patients, one was not evaluable because the patient had not had the first tumor assessment at the time of the data cut-off. The median age is 69 (41-81) and 70.6% were male, with 1 (0-3) as the median prior lines of treatment. 41.2% of 17 patients were non-smokers and 58.8% were smokers. ABN401 continued to demonstrate outstanding safety compared to the currently marketed drugs.

The results were presented in a poster session at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in Boston, and at a separate company event nearby.

"We are delighted to see the outstanding data from the global Phase 2 clinical trial to evaluate this potential best-in-class treatment, which continues to exhibit an outstanding safety profile," said Young Kee Shin, M.D., Ph.D., CEO of ABION. "Notably, these results follow and further expand the potential of ABN401 combination therapy with other treatment regiments. "The excellent safety enables the potential combination of ABN401 with EGFR treatment, chemotherapy, or KRAS or immuno-oncology treatments. Our goal is to increase options for patients with c-MET positive NSCLC."

Based on these results, ABION plans to initiate a Phase 2 combination trial with a multiple drug regimen. The c-MET mutation is the most common mutation shown by NSCLC patients after treatment with EGFR TKIs.

InterVenn Biosciences Announces the Publication of Early Detection of Advanced Adenoma and Colorectal Cancer in Gastroenterology, the Flagship Journal of the American Gastroenterological Association

On October 13, 2023 InterVenn Biosciences, a life sciences company pioneering glycoproteomics, reported the publication of their discovery work in Advanced Adenoma (AA) and Colorectal Cancer (CRC) early disease detection in the flagship journal of the American Gastroenterological Association, Gastroenterology (Press release, InterVenn Biosciences, OCT 13, 2023, View Source [SID1234635962]). This groundbreaking research in early detection of CRC represents an advancement in early diagnosis through serum glycoproteome profiling. InterVenn’s GlycoVision platform leverages advanced liquid chromatography-mass spectrometry (LC-MS) combined with artificial intelligence (AI) and neural networks (NN) to interrogate a new layer of biology, enabling novel insights and discoveries.

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The results published in Gastroenterology establish the groundwork for a blood-based CRC screening modality using artificial intelligence to reveal the earliest stages of colorectal cancer. The proprietary LC-MS and AI/NN platform, GlycoVision, has been developed to accelerate data analysis of biological information that has not previously been practical or efficient.

The research involved a comprehensive case-control analysis using well-annotated blood samples from 279 cases (CRC and advanced adenomas) and 296 control samples. InterVenn’s sophisticated LC-MS-based glycoproteomic quantification workflow was employed, resulting in the development of a novel multivariable classifier model using six key biomarkers, which, when applied to a test set, achieved an Area under the Receiver Operating Characteristic curve (AuROC) of 0.96, signifying notable sensitivity and specificity in detecting both CRC and advanced adenoma. The classifier exhibited a sensitivity of 90.9% for advanced adenoma without high grade dysplasia, 85.7% for Advanced Adenoma with High Grade Dysplasia and 89.8% for all stages of CRC, including 91.2% for early-stage (stage 1 and 2) CRC, and 89.4% for late-stage (stage 3 and 4) CRC, along with high specificity of 89% for normal findings.

"InterVenn’s novel AI-powered glycoprotein-based strategy is among the most differentiated and compelling value propositions in the diagnostic industry," said Josh Stahl, InterVenn’s recently appointed CEO. "The resulting performance of the GlycoVision platform has the potential to aid patients, providers, and payers in the early diagnosis of colorectal cancer."

"This collaborative study, conducted in partnership with some of the world’s foremost experts in colorectal cancer, represents a pivotal milestone in advancing the development of an innovative blood-based CRC screening test," stated Daniel Hommes, gastroenterologist and Chief Medical Officer.

Details on the Publication:

Desai K, Gupta S, May FP, Xu G, Shaukat A, Hommes DW, NICE-AA/CRC- Consortium, Early detection of advanced adenomas and colorectal carcinoma by serum glycoproteome profiling., Gastroenterology (2023), doi: View Source