On September 16, 2021, Silverback Therapeutics, Inc. (the "Company") presented its corporate slide presentation (Presentation, Silverback Therapeutics, SEP 16, 2021, View Source [SID1234587805]).

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On September 16, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported results of a new study showing that local treatment in vivo with INTASYL can cure tumors and generate systemic tumor immunity that is both durable and tumor specific (Press release, Phio Pharmaceuticals, SEP 16, 2021, View Source [SID1234587804]). These data, which were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021, highlight the potential of the Company’s INTASYL technology in direct therapeutic applications.

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The study was performed to show the synergistic activity of co-targeting PD-1 and BRD4 with one INTASYL formulation (PH-3861) in a preclinical in vivo hepatocellular carcinoma model. The results show that up to 83% of the animals treated with PH-3861 had a complete response when treated at low doses, namely doses suboptimal for monotherapy. Moreover, this treatment induced a durable and specific systemic anti-tumor immune response, without requiring further treatment.

"We continue to build on data presented earlier this year and show how our INTASYL technology can be utilized in various applications to treat cancer, including its broad applicability as a direct therapeutic. Since cancer is a multifactorial disease, being able to address multiple factors at once is a significant benefit. The data presented today at the ESMO (Free ESMO Whitepaper) Congress demonstrate that we can target multiple genes in one INTASYL product, resulting in powerful therapeutics showing complete resolution of tumors and generation of tumor-specific immunity that persists for months after the initial treatment," said Dr. Simon Fricker, Phio’s Vice President of R&D. "We look forward to further validate INTASYL as a therapeutic modality in an upcoming clinical trial with our lead INTASYL product candidate."

In the study presented today, subcutaneous Hepa1-6 tumors in mice were treated with a murine version of dual targeting INTASYL PH-3861 (mPH-3861) by local administration to the tumor, at low doses that are suboptimal for a single agent targeting either PD-1 or BRD4 alone. Mice showing complete cure of tumors with mPH-3861 were rechallenged by implanting hepatoma cancer cells at a different location to the previous tumor challenge up to 2.5 months after the initial treatment. All of the rechallenged mice previously cured by mPH-3861 were cured again without requiring further treatment, while tumors grew steadily in the control group as expected. These data show that treatment with mPH-3861 provides a durable and systemic anti-tumor immune response that can combat tumor growth.

A poster further detailing the data presented at the ESMO (Free ESMO Whitepaper) Congress 2021 titled "Dual targeting PD-1 and BRD4 with INTASYL self-delivering RNAi elicits complete resolution of tumors and persistent anti-tumor immunity in vivo" will be made available on the "Investors – Events and Presentations" section of the Company’s website (click here).

On September 16, 2021 MacroGenics, Inc. (NASDAQ: MGNX), a biopharmaceutical company focused on developing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported results from Cohort A Part 1 of the Phase 2/3 MAHOGANY clinical trial of margetuximab (Press release, MacroGenics, SEP 16, 2021, View Source [SID1234587802]). MARGENZA (margetuximab-cmkb) is approved in HER2+ metastatic breast cancer and is being investigated as a potential first-line treatment for patients with HER2+ gastric cancer (GC) or gastroesophageal junction (GEJ) cancer in combination with a checkpoint inhibitor, with or without chemotherapy. The dataset is available in a poster titled "Margetuximab With Retifanlimab in HER2+, PD-L1+ First-Line Unresectable/Metastatic Gastroesophageal Adenocarcinoma (GEA): MAHOGANY Cohort A" (Poster #1379P) at the 2021 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Conference taking place September 16-21, 2021.

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The efficacy data and safety cutoff dates were July 19, 2021 and August 3, 2021, respectively. In Cohort A Parts 1 and 2, the efficacy and safety of combining margetuximab and retifanlimab (investigational anti-PD-1 monoclonal antibody licensed to Incyte by MacroGenics) is planned to be evaluated in approximately 100 patients whose tumors are HER2+ at the 3+ level by immunohistochemical (IHC) staining, PD-L1+ (combined positive score ≥1%) and non-microsatellite instability-high (non-MSI-H). A pre-specified interim analysis assessing efficacy and safety was conducted on the first 40 non-MSI-H patients enrolled in Part 1. These data support advancement to Part 2 with plans to enroll approximately 60 additional response-evaluable non-MSI-H patients.

A total of 43 HER2 3+ and PD-L1+ patients were enrolled in Cohort A Part 1 and received margetuximab 15 mg/kg plus retifanlimab 375 mg/kg administered intravenously every three weeks. Twenty-five patients (58%) had gastric cancer and 18 patients (42%) had gastroesophageal junction cancer; 36 patients (84%) had metastatic disease at study entry.

MAHOGANY Cohort A Interim Analysis

Anti-tumor activity was observed in patients treated with margetuximab plus retifanlimab in MAHOGANY Cohort A after the first scan. Tumor shrinkage was observed in 32 of 41 patients (78%) with at least one post-baseline target lesion measurement. Twenty-one of 40 response-evaluable patients achieved an objective response (53%, 95% confidence interval (CI): 36%-69%), including four confirmed complete responses and 17 confirmed partial responses. The number of confirmed responders by independent assessment exceeded the prespecified futility boundary for the trial, and enrollment is proceeding to Cohort A Part 2.

Disease control was achieved in 29 of 40 patients (73%, CI: 56%-85%) and the median duration of response was 10.3 months (range: 2.1 – 14.5 months, CI: 4.6 months – not evaluable (NE)). Median progression-free survival (PFS) was 6.4 months by independent assessment (CI: 6.0 months – NE); median overall survival (OS) was not yet reached. At both 12 and 18 months, OS was 85% (CI: 63%-95%).

Antitumor activity was comparable to historical data from the experimental arm of the Trastuzumab for Gastric Cancer (ToGA) study (trastuzumab + chemotherapy; n=294; objective response rate (ORR) of 47%; median duration of response (DOR) of 6.9 months)1 and initial data from the control arm (placebo + trastuzumab + chemotherapy) of the KEYNOTE‐811 study (ORR of 52%; median DOR of 9.5 months).2

The safety analysis of all 43 patients treated with margetuximab plus retifanlimab suggests the combination was well tolerated in the study population. The most common TRAEs were fatigue (21% Grade 1-2, 0% Grade ≥3), infusion-related reaction (19% Grade 1-2, 0% Grade ≥3), rash (19% Grade 1-2, 0% Grade ≥3), diarrhea (16% Grade 1-2, 2% Grade 3), and pruritus (16% Grade 1-2, 0% Grade ≥3). A total of nine Grade 3 TRAEs were reported in eight patients (19%); no Grade 4 TRAEs were observed. Eight serious TRAEs were reported in seven patients. Infusion-related reactions considered as adverse events (AEs) of special interest occurred in six patients.

Treatment-emergent AEs of Grade 3 occurred in 18 of 43 patients (42%) of patients. Three of 43 patients (7%) discontinued therapy due to immune-related AEs: renal dysfunction (Grade 3), hepatitis (Grade 3), and diabetic ketoacidosis (Grade 1); no AEs led to death.

Safety data from MAHOGANY compare favorably to the experimental arm of ToGA in which overall Grade 3-4 AEs were 68% and the treatment-related mortality was 3%.1 Initial results from KEYNOTE-811 data presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting indicated that AEs of Grade 3-5 occurred in 57.1% of patients in the experimental arm (pembrolizumab + trastuzumab + chemotherapy) and in 57.4% of patients in the control arm, AEs leading to death occurred in 3.2% vs 4.6%, and AEs leading to discontinuation of any study drug occurred in 24.4% vs 25.9% of patients, respectively. Despite limitations of cross-study comparisons, regimens containing chemotherapy may have clinically relevant safety differences compared to the chemotherapy-free regimen in MAHOGANY Cohort A.

"We are excited to share our results from the interim analysis of Part 1 of the MAHOGANY Cohort A study at ESMO (Free ESMO Whitepaper)," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "This study is designed to support potential registration of margetuximab in combination with other agents for patients with gastric or gastroesophageal junction cancer as part of our strategy to advance margetuximab in HER2+ cancer. The findings suggest the combination of margetuximab and retifanlimab may potentially provide a chemotherapy-free option as a first-line treatment for patients whose tumors are positive for both HER2 and PD-L1. We are pleased these data support the protocol’s prespecified advancement into Part 2 of MAHOGANY Cohort A. We plan to discuss these results and future development of the combination in an upcoming scheduled meeting with the FDA."

ESMO Presentation
MacroGenics’ Cohort A Part 1 MAHOGANY Study poster presentation is available for on-demand viewing on the ESMO (Free ESMO Whitepaper) website and on the "Events & Presentations" page on MacroGenics’ website at View Source

The MAHOGANY Study Design

MAHOGANY (NCT04082364) is a Phase 2/3 clinical trial in two cohorts designed to evaluate margetuximab in combination with a checkpoint inhibitor, with or without chemotherapy, as a potential first-line treatment for patients with advanced or metastatic HER2+ GEJ/GC.

Cohort A is designed as a single arm study to test margetuximab plus retifanlimab (previously known as MGA012 and INCMGA00012), an investigational anti-PD-1 monoclonal antibody, in patients with HER2+ and PD-L1+ tumors. The primary outcome measure for efficacy is ORR per Response Evaluation Criteria in Solid Tumors (RECIST 1.1).

Cohort B is designed as a randomized trial to test margetuximab plus a checkpoint inhibitor in combination with chemotherapy compared to standard of care therapy of trastuzumab with chemotherapy in patients with HER2+ tumors irrespective of PD-L1 expression. Patients randomized to one of two experimental arms containing a checkpoint inhibitor will receive either retifanlimab or tebotelimab (previously known as MGD013), an investigational DART molecule targeting PD-1 and LAG-3. The primary outcome measure for efficacy is OS.

The Phase 2/3 clinical trial is being conducted at clinical sites globally, in collaboration with Zai Lab, the company’s regional partner in Greater China. For additional information about the MAHOGANY study, please visit View Source

About Gastric and Gastroesophageal Junction Cancer

Cancer of the stomach (gastric cancer, GC), or the gastroesophageal junction (GEJ) where the esophagus joins the stomach, is collectively known as gastroesophageal adenocarcinoma and is the fifth most common tumor type worldwide. Both GC and GEJ cancer are often diagnosed at an advanced stage and therefore have very poor prognosis, with a 5-year survival of 5-20%. Chemotherapy is the standard of care for first-line therapy and may be combined with trastuzumab for the approximately 20% of patients whose tumors are HER2+.

About Margetuximab

MARGENZA (margetuximab-cmkb) is approved in HER2+ metastatic breast cancer and is being investigated as a potential first-line treatment for patients with HER2+ GC or GEJ cancer in combination with a checkpoint inhibitor, with or without chemotherapy. Margetuximab is an Fc-engineered monoclonal antibody that targets the HER2 oncoprotein. HER2 is expressed by tumor cells in breast, gastroesophageal and other solid tumors. Through MacroGenics’ Fc Optimization technology, margetuximab has been engineered to enhance the engagement of the immune system.

Margetuximab is also being evaluated in combination with tebotelimab (PD-1 × LAG-3 bispecific DART molecule) in various HER2+ tumors (NCT03219268). MacroGenics is partnered with Zai Lab for the development and commercialization of margetuximab in Greater China. For more information, please visit www.clinicaltrials.gov.

About Retifanlimab

Retifanlimab is an investigational, humanized, proprietary anti-PD-1 monoclonal antibody being developed for use as monotherapy as well as in combination with other potential cancer therapeutics. Retifanlimab was licensed to Incyte Corporation in 2017 under an exclusive global collaboration and license agreement. MacroGenics retains the right to develop its pipeline molecules with retifanlimab. Incyte is pursuing development of retifanlimab monotherapy in four potentially registration-directed trials for patients with MSI-high endometrial cancer, Merkel cell carcinoma, anal cancer and non-small cell lung cancer. Incyte and MacroGenics are each conducting multiple studies of retifanlimab in combination with other agents.

About Tebotelimab

Tebotelimab is an investigational, first-in-class bispecific DART molecule designed to provide co-blockade of PD-1 and LAG-3 for the potential treatment of a range of solid tumors and hematological malignancies.

On September 16, 2021 MacroGenics, Inc. (NASDAQ: MGNX), a biopharmaceutical company focused on developing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported preliminary safety and anti-tumor activity data from dose expansion cohorts of the Company’s ongoing Phase 1 clinical trial of MGC018 (Press release, MacroGenics, SEP 16, 2021, View Source [SID1234587801]). This investigational antibody-drug conjugate (ADC) was designed to deliver a DNA-alkylating duocarmycin payload to both dividing and non-dividing cells in a B7-H3-dependent manner. The dataset is being presented in a poster titled "MGC018, an Anti-B7-H3 Antibody-Drug Conjugate (ADC), in Patients with Advanced Solid Tumors: Preliminary Results of Phase 1 Cohort Expansion" (Poster #620P) at the 2021 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Conference taking place September 16-21, 2021.

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Cohort Expansion Results Update

As of the August 16, 2021 data cut-off, a total of 86 patients with advanced solid tumors were enrolled in the cohort expansion of MGC018 at the recommended Phase 2 dose (RP2D) of 3.0 mg/kg, administered intravenously every three weeks. The enrollment includes 40 patients with mCRPC, 21 patients with NSCLC, 16 patients with triple negative breast cancer (TNBC) and nine patients with melanoma. In addition, enrollment of patients with squamous cell carcinoma of the head and neck (SCCHN) was recently initiated. The safety analysis both in the poster and below includes all enrolled patients, whereas the efficacy analysis was limited to mCRPC and NSCLC patients, as enrollment continues in the other tumor cohorts.

In the cohort expansion, tumor response by investigator per Response Evaluation Criteria in Solid Tumors (RECIST v1.1) was evaluated every nine weeks for all patients and PSA was assessed every three weeks in mCRPC.

Preliminary Anti-tumor Results for mCRPC Cohort Expansion

As of the August 16, 2021 data cut-off, all 40 patients in the mCRPC cohort expansion had been enrolled. Patients had previously received a median of three prior therapies for advanced disease, with all 40 patients having received both chemotherapy and next-generation hormonal therapy. The median B7-H3 H-score for all mCRPC patients was 223.

A total of 39 mCRPC patients were evaluable for PSA response. Reductions in PSA levels of ≥ 50% were observed in 21 of 39 patients (54%). Twenty-four of the 39 patients (62%) remained on treatment as of the data cut-off.

Of the 40 patients in the mCRPC cohort, 16 of the 23 patients with measurable disease were evaluable for tumor response by RECIST as of the data cut-off. Ten of these 16 patients (63%) had reductions in their target lesion sums from baseline. Four patients (25%) demonstrated a partial response (PR), consisting of two confirmed and two unconfirmed PRs. Treatment was ongoing in six of 16 patients with evaluable tumor response as of the data cut-off.

Preliminary Anti-tumor Results for NSCLC Cohort Expansion

As of the August 16, 2021 data cut-off, the NSCLC cohort expansion had been fully enrolled with 21 patients. Patients had previously received a median of two prior therapies for advanced disease, with 15 (71%) having previously received anti-PD-1/PD-L1 therapy. The median B7-H3 H-score for these patients was 139.

A total of 16 NSCLC patients were evaluable for tumor response by RECIST. Thirteen of 16 (81%) patients had reductions in their target lesion sums from baseline. Four of these 16 patients (25%) experienced unconfirmed partial responses. Another one of these 16 patients experienced a 30% reduction in target lesions; however, the patient’s non-target lesions were not evaluated due to an obstruction of the bronchus and overall response was not evaluable. Treatment was ongoing in seven of 16 patients as of the data cut-off.

Preliminary Safety Results

The safety analysis includes all 86 patients enrolled in the cohort expansion as of the August 16, 2021 data cut-off. The median number of doses received by mCRPC patients was 3.5 (range: 1-8); those with NSCLC received 3.0 (range: 1-7). Adverse events for the dose expansion cohorts of 3 mg/kg were generally consistent with those previously reported at ASCO (Free ASCO Whitepaper) 2021. TRAEs included hematologic and skin toxicities that have been clinically manageable to date. In the cohort expansion study overall, at least one TRAE of any grade was experienced by 78 of 86 patients (91%), with 43 of 86 patients (50%) experiencing a Grade ≥3 TRAE. There were two Grade 5 fatal events: one from an unknown cause and one due to SARS-CoV-2.

The most common TRAEs were fatigue (37% all grades; 1% Grade ≥3), neutropenia (34% all grades; 22% Grade ≥3), palmar plantar erythrodysesthesia syndrome (31% all grades; 4% Grade ≥3), pleural effusion (23% all grades; 1% Grade ≥3), nausea (22% all grades; 1% Grade ≥3), asthenia (20% all grades; 5% Grade ≥3) and thrombocytopenia (14% all grades; 7% Grade ≥3). The overall results have demonstrated a manageable safety profile with a low rate of treatment discontinuation due to TRAEs: only six of 86 (7%) patients had discontinued therapy in the cohort expansion as of the data cut-off date due to TRAEs.

"We are highly encouraged by the growing data from our ongoing Phase 1 study of MGC018," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "Consistent with previously presented data, we observed PSA reductions of 50% or greater in 54% of patients with metastatic castration-resistant prostate cancer. And now, we are particularly pleased to see partial responses emerge – both confirmed and unconfirmed – in mCRPC and NSCLC patients, with encouraging anti-tumor activity observed in the majority of patients for both tumor types. Also, we are very pleased with the evolving safety profile of MGC018, which showed neutropenia as the only Grade 3 or higher TRAE that exceeded a rate of 10% in this trial. Finally, further optimization of patient management has resulted in a low rate of MGC018 discontinuation due to TRAEs as of the data cutoff. Enrollment is ongoing in the TNBC, SCCHN, and melanoma cohorts and we look forward to providing further updates on patients in these cohorts, as well as patients in the mCRPC and NSCLC cohorts, at subsequent scientific conferences."

ESMO Presentation

MacroGenics’ MGC018 poster presentation is available for on-demand viewing on the ESMO (Free ESMO Whitepaper) website and on the "Events & Presentations" page on MacroGenics’ website at View Source

About MGC018

MGC018 is an ADC comprised of an anti-B7-H3 humanized IgG1/kappa monoclonal antibody conjugated via a cleavable linker to the prodrug seco-DUocarmycin hydroxyBenzamide Azaindole (DUBA; licensed from Byondis, B.V.), with an average drug-to-antibody ratio (DAR) of ~2.7. DUBA is an alkylating agent that can damage DNA in both dividing and non-dividing cells, causing cell death. B7-H3 is a molecule highly expressed on many solid tumors and associated with a poor clinical outcome. MGC018 is being evaluated in a Phase 1 study (NCT03729596). MacroGenics retains worldwide rights to MGC018.

On September 16, 2021 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported that the U.S. Food and Drug Administration (FDA) has accepted the investigational new drug (IND) application for NTLA-5001, the company’s first wholly-owned ex vivo CRISPR genome editing candidate for the treatment of cancer (Press release, Intellia Therapeutics, SEP 16, 2021, View Source [SID1234587800]). NTLA-5001 is an autologous T cell receptor (TCR)-T cell therapy engineered to target the Wilms’ Tumor (WT1) antigen for the treatment of all genetic subtypes of acute myeloid leukemia (AML). Intellia intends to initiate patient screening by year-end in a Phase 1/2a study evaluating NTLA-5001 in adults with persistent or recurrent AML who have previously received first-line therapy.

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"The FDA’s acceptance of our IND for NTLA-5001 is an important milestone in our pursuit of developing advanced cell therapies utilizing Intellia’s proprietary engineering platform to treat patients with cancer. NTLA-5001 is our first wholly-owned ex vivo candidate to enter the clinic, and we expect to initiate this first-in-human study in adults with AML by year-end. Our treatment strategy is to leverage CRISPR/Cas9 genome editing technology to create next-generation engineered immune cells with the potential to attack cancer cells more effectively and safely than previously developed cell therapies," said Intellia President and Chief Executive Officer John Leonard, M.D. "Our study is an important first step toward improving treatment for people living with this aggressive form of cancer. AML is the most common type of acute leukemia in adults, that, despite currently available treatments, has a five-year survival rate of less than 30 percent."

The Phase 1/2a study will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults who have detectable AML after having received standard first-line therapy. The study will contain a dose escalation and expansion phase, with up to 54 participants. The dose-escalation phase of the study will include two independent arms of up to three cohorts: Arm 1 will consist of adults with AML with lower disease burden, defined as those with less than 5% AML blasts in bone marrow, while Arm 2 will consist of adults with AML with higher disease burden, defined as those greater than or equal to 5% AML blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment. More information about the study will be available at clinicaltrials.gov.

In addition to the U.S., Intellia has also submitted a regulatory application to the U.K. for NTLA-5001.

About Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment. It is the most common type of acute leukemia in adults, with more than 20,000 estimated new cases in 2020. Despite currently available treatments for AML, the five-year overall survival rate for patients remains less than thirty percent. AML, along with other cancer types, is often characterized by overexpression of the Wilms’ Tumor 1 (WT1) antigen.

About NTLA-5001

NTLA-5001 is a CRISPR/Cas9-engineered T cell receptor (TCR)-T cell therapy in development for the treatment of all genetic subtypes of acute myeloid leukemia (AML). This autologous cell therapy candidate is designed for AML patients with the HLA-A*02:01 allele whose tumors carry the Wilms’ Tumor 1 (WT1) antigen, which is widely overexpressed in AML and other cancers. NTLA-5001 is Intellia’s first wholly-owned ex vivo therapeutic candidate, developed using its proprietary cell engineering platform for the treatment of cancer. Based on preclinical results, Intellia believes its proprietary cell engineering platform will result in a pipeline of more efficacious and safer cell-based cancer therapies.