Myeloid Therapeutics Presents Late-Breaking Data at AACR 2022 Demonstrating the Ability of its ATAK™ Receptors and in vivo Delivery of mRNA to Attack Cancer by Immune Reprogramming

On April 10, 2022 Myeloid Therapeutics, Inc. ("Myeloid"), a clinical stage mRNA-immunotherapy company developing novel therapies for cancer and autoimmune diseases, reported that new late-breaking data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, being held in New Orleans, LA, April 8-13, 2022 (Press release, Myeloid Therapeutics, APR 10, 2022, View Source [SID1234611861]).

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The data presented at AACR (Free AACR Whitepaper) 2022 illustrate that Myeloid has designed and developed two novel therapeutic platforms, ATAK CAR receptors and in vivo mRNA programming, to target and activate the ability of myeloid cells to attack cancer by immune reprogramming. Myeloid cells are a primary orchestrator of immune response and accumulate naturally within solid tumors, in some cases representing up to seventy-five percent of the tumor mass. Myeloid’s adaptations of mRNA for the myeloid compartment are expanding the impact of these cells within in vivo experiments.

Myeloid’s novel class of CARs, known as ATAK Receptors, combine tumor recognition with multiple proprietary innate-immune signaling domains. Myeloid scientists have screened multiple unexplored combinations of innate-immune signals and uncovered optimal multi-signal pathways. The combination of cancer recognition binders with these novel intracellular signaling domains allows myeloid cells to be reprogrammed with previously unexplored combinations of immune signals, leading to tumor killing and broad systemic anti-tumor responses.

Myeloid’s novel in vivo engineering platform specifically targets and activates myeloid cells to elicit broader anti-tumor adaptive immunity. Through this approach, Myeloid demonstrates that delivery of lipid-nanoparticles (LNPs) encapsulating mRNA results in selective uptake and expression by myeloid cells in vivo, leading to potent tumor killing in multiple cold tumor models. These data demonstrate the potential for Myeloid’s technology to program cells directly in vivo.

"At this year’s AACR (Free AACR Whitepaper) meeting, we are pleased to present significant progress across our platforms that showcase the ability of myeloid cells to orchestrate broad immune responses through in vivo mRNA programming and our next-generation ATAK CARs," said Bruce McCreedy, Ph.D., Chief Scientific Officer of Myeloid. "These data support our plans to initiate clinical trials to evaluate the safety and activity of several novel drug product candidates within the next year, expanding our existing clinical pipeline."

Details of the late-breaking poster presentations:

Title: "In vivo programming of myeloid cells by mRNA-mediated delivery of novel Fc alpha fusion receptor activates anti-tumor immunity"
Session: Late-Breaking Research: Clinical Research 1
Date and Time: Sunday Apr 10, 2022 1:30 PM – 5:00 PM
Location: New Orleans Convention Center, Exhibit Halls D-H, Poster Section 17

Title: "ATAK receptors, a new class of chimeric antigen receptors that harness innate immunity in myeloid cells to target cancer"
Session: Late-Breaking Research: Clinical Research 1
Date and Time: Sunday Apr 10, 2022 1:30 PM – 5:00 PM
Location: New Orleans Convention Center, Exhibit Halls D-H, Poster Section 17

Abstracts and full session details can be accessed through the AACR (Free AACR Whitepaper) meeting planner once available: AACR (Free AACR Whitepaper) Annual Meeting 2022 | April 8-13, 2022 | New Orleans

Telix Pharmaceuticals Announces Licence Agreement with Lilly for Olaratumab

On April 10, 2022 Telix Pharmaceuticals Limited (ASX: TLX, "Telix", the "Company") reported that it has entered into a licence agreement with Eli Lilly and Company ("Lilly") under which Telix is granted exclusive worldwide rights to develop and commercialise radiolabelled forms of Lilly’s olaratumab antibody for the diagnosis and treatment of human cancers (Press release, Telix Pharmaceuticals, APR 10, 2022, View Source [SID1234611860]). Telix’s initial development focus will be on a rare type of cancer known as soft tissue sarcoma (STS).

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Olaratumab was originally developed by Lilly as a (non-radiolabelled) monoclonal antibody targeting Platelet Derived Growth Factor Receptor Alpha (PDGFRα). PDGFRα is expressed in multiple tumour types including STS. STS is generally a radiation susceptible cancer that may be inherently amenable to systemic radionuclide therapy and olaratumab’s ability to target PDGFRα makes it a highly novel and potentially exciting candidate for use as a radionuclide targeting agent. The exclusive worldwide licence will allow Telix to repurpose olaratumab as a targeting agent for radiopharmaceutical imaging and therapy of cancer. Olaratumab has an established safety profile that underpins its potential use as a radionuclide targeting agent.

Material terms of the agreement
Under the terms of the agreement Telix will pay Lilly an upfront payment of US$5M (~AU$6.7M) for the grant of an exclusive licence to Lilly’s intellectual property related to the development of a radiolabelled olaratumab, as well as access to material for use by Telix in initial pre-clinical and early-phase clinical studies in application to potential uses for the diagnosis and treatment of human cancers.

Lilly may be eligible for up to US$225M (~AU$301M) in payments based upon the achievement of pre-specified development, regulatory and commercial milestones. Lilly would also be eligible to receive industry standard royalties on net sales. The agreement also includes an option for Lilly to be granted an exclusive licence to a radiolabelled companion diagnostic which would be developed by Telix. If exercised, Lilly will pay Telix US$5M (~AU$6.7M) and up to US$30M (~AU$40.1M) in potential development milestones, as well as industry standard royalties.

The agreement has typical termination rights for breach and related corporate issues. Telix retains termination rights typical to licence agreements of this nature to enable the Company to exit the agreement based on a development or commercial basis.

Building on Telix’s track record in acquiring and commercialising assets
Telix Group CEO and Managing Director, Dr. Christian Behrenbruch said, "This in-licence transaction with Lilly is a valuable – and rare – opportunity to acquire an asset which has demonstrated clinical safety. In our pre-transaction diligence and research, we have identified that a radiolabelled version of olaratumab could be efficacious in patients with STS, particularly as it is a highly radiation-sensitive cancer. The safety data generated by Lilly in relation to the original development program significantly de-risks the program for Telix. We anticipate that early clinical translation with a radiolabeled olaratumab as an imaging agent may also provide valuable clinical information as to whether this asset has potential therapeutic efficacy, demonstrating the advantage of Telix’s "theranostic" approach.

"This acquisition mirrors the approach that Telix has taken in building its existing pipeline by in-licencing or acquiring assets that have already been proven to be safe for use in humans that can be harnessed as novel radiolabelled targeting agents. This partnership also demonstrates the value that Telix can bring as a capable partner with the expertise in radiopharmaceutical development and manufacturing, to help repurpose or expand the use of promising candidates to better target, find and treat cancer."

About Soft Tissue Sarcoma (STS)
Soft tissue sarcoma is a complex disease that encompasses a diverse group of relatively rare cancers, with more than 50 histological subtypes. In the United States, it is estimated that 13,040 new cases and 5,150 deaths were caused by STS in 2019, representing 0.75% of overall cancer incidence and 0.84% of overall cancer mortality.1 In Europe, nearly 23,600 new STS cases rose annually, and the crude incidence rate was 4.7 per 100,000.2 Approximately 39,900 new STS cases occurred nationwide in China in 2019, accounting for 1.05% of overall cancer incidence.3 The crude incidence rate was 2.91/100,000 and generally increased with age. Standard treatment for soft tissue sarcoma includes surgery, radiation therapy and/or chemotherapy. For patients with advanced, unresectable, or metastatic disease, treatment typically involves chemotherapy with single agents (e.g., doxorubicin) or anthracycline-based combination regimens. However, the prognosis for these patients remains poor, with treated patients with metastatic disease having a median overall survival of around 12–18 months.

About olaratumab
Olaratumab (previously sold under the brand name, Lartruvo) was originally developed as a monoclonal antibody targeting PDGFRα. Olaratumab was granted "Accelerated Approval" in the US and "Conditional Approval" in the EU based on Phase 2 trial data which showed a 1-year survival benefit in patients with STS, when given in combination with standard chemotherapy. Olaratumab was voluntarily withdrawn from the market by Lilly following the failure of the Phase 3 ANNOUNCE clinical trial, in which olaratumab did not improve survival for patients.

Innovent Release Phase 1 Results of IBI322 (anti-PD-L1/CD47 bispecific antibody) in patients with advanced solid tumors at AACR Annual Meeting 2022

On April 10, 2022 Innovent Biologics, Inc. ("Innovent", HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, reported that the results of the Phase I study for IBI322 (anti-CD47 /PD-L1 bispecific antibody) in patients with advanced solid tumors were presented today at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 (Poster No. CT513) (Press release, Innovent Biologics, APR 10, 2022, View Source [SID1234611859]).

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This is a first-in-human, phase Ia/Ib dose escalation/expansion study of IBI322 monotherapy in patients with advanced solid tumors who failed standard of care treatment. 58 subjects were enrolled in the study, 16 of which (27.6%) had previously received PD-1/L1 treatment. The highlights for the study results were as follows:

20 patients with various advanced solid tumors at active doses of IBI310 ≥10 mg/kg group were evaluable per RECISTv1.1, 4 achieved PR, with investigator assessed ORR 20%.
Among 9 patients with NSCLC treated at active doses of IBI310 ≥10 mg/kg group, 3 achieved PR, with investigator assessed ORR 33.3% and the DCR 88.9%.
IBI322 was well tolerated and showed a favorable safety profile. Treatment related adverse events (TRAEs) occurred in 74.1% (43/58) patients, most frequent TRAEs including anemia, platelet count decreased, pyrexia. The majority of the TRAEs were in grade 1-2. No treatment related death occurred as the cut-off date.
Phase Ia dose expansion study with IBI322 monotherapy has kept updating with longer follow-up after the cut-off date. Preliminary response and disease control have been observed in specific indications (such as SCLC). More clinical data will be mature and presented in the future.

Furthermore, given that IBI322 has demonstrated promising efficacy signals and favorable safety and tolerability profile, phase Ib trial has been conducted to further explore the safety and efficacy of IBI322 in multiple indications.

Professor Jie Wang, principal investigator of the study, Chief of Department of Medical Oncology, Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, stated: "Immune checkpoint inhibitors (ICI) have shown promising efficacy in various tumor types, but many clinical challenges still remain. With the increasing prescription of ICI in first-line setting, many patients developed into ICI resistance or has poor response rate in clinical practice. Therefore, it is of great clinical significance to develop next generation bi-specific immune checkpoint inhibitors. CD47 is one of the most promising targets in immunotherapy. With innovate bispecific antibody development technology, IBI322 showed favorable safety and preliminary efficacy signals in subjects who had failed previous standard treatment, which increased our confidence in the subsequent expansion cohort study."

Dr. Hui Zhou, Vice President of Innovent, stated: "IBI322 is a bispecific antibody that specifically targeting recombinant anti-differentiation cluster 47 (CD47) and anti-programmed death ligand 1 (PD-L1).IBI322 is independently developed by Innovent Biologics and the company owns global proprietary rights. We are very excited to observe preliminary efficacy and manageable safety profile in patients with advanced malignancy who failed standard of care treatment. We will continue to proceed the phase Ib expansion cohort study and further explore the safety and efficacy of IBI322 in multiple indications. By developing a comprehensive and advanced pipeline of next generation immune checkpoint inhibitors, we hope to bring clinical benefit to more patients."

About IBI322

IBI322 is a recombinant anti-human CD47/PD-L1 bispecific antibody developed by Innovent Biologics. As a bispecific antibody, IBI322 targets CD47 on the surface of tumor cells, blocks SIRPα/CD47 pathway and activates macrophages to attack the tumor cells. Furthermore, IBI322 target PD-L1 on the surface of tumor cells, blocks the PD-1/PD-L1 pathway, which counteracts the inhibition of T cells and activates the T cells to attack the tumor cells. By inhibiting two different targets, IBI322 can not only activate both innate immune pathway and adaptive immune pathway, which provides synergistic effect, but also reduce the red blood cell destruction. IBI322 has received IND approvals from both the NMPA and the U.S. FDA and has been actively developed globally.

Antengene Announces Publication of Five Posters at the 2022 American Association for Cancer Research (AACR) Annual Meeting

On April 10, 2022 Antengene Corporation Limited ("Antengene" SEHK: 6996.HK), a leading innovative, global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class medicines for cancer, reported the publication of five posters that will be presented during the upcoming 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (AACR 2022), taking place from April 8th to April 13th in New Orleans in person or via virtual attendance (Press release, Antengene, APR 10, 2022, View Source [SID1234611858]).

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"The preclinical studies that we are presenting at AACR (Free AACR Whitepaper) 2022 provide a window into five innovative programs in Antengene’s pipeline," said Bo Shan, Ph.D., Chief Scientific Officer of Antengene. "These programs target areas that we believe are very important in cancer drug development: Tumor microenvironment (TME) regulators (ATG-037), pathway inhibitors (ATG-018, ATG-022 and ATG-008), and ADCs (ATG-022). These studies have been instrumental in guiding our clinical development plans for each program, including selection of combination partners and biomarkers, that could be used to predict efficacy or improve the proportion of patients who respond to treatment. We are very pleased to share these results with the oncology community."

Details of the posters and corresponding abstracts are shown below:

ATG-037, a highly potent small molecule CD73 inhibitor has superior activity of reversing immunosuppression in higher-AMP environments compared with anti-CD73 antibodies

Abstract: 2576

Session: Cell Cycle, Replication Inhibitors, and Immunotherapy Agents

Date and Time: 9:00 AM – 12:30 PM CST, April 12, 2022

Venue: Poster Section 21

This study was designed to compare the T-cell rescue activity of ATG-037, a highly potent and selective oral small molecule inhibitor of CD73, and two CD73 blocking antibodies. CD73 is an enzyme that is highly expressed in the tumor microenvironment and enables the degradation of AMP into adenosine, resulting in immunosuppression and cancer progression. In vitro assays were used to assess each compound’s ability to inhibit CD73 enzyme activity and reverse AMP/adenosine mediated T-cell suppression. ATG-037 demonstrated more potent and complete inhibitory activity of cell surface CD73 in this study.

As shown in Figure, the authors found that ATG-037 had a stronger ability to restore T-cell function in higher-AMP environments compared with other clinical anti-CD73 antibodies. These data highlight the potential therapeutic advantages of small molecule inhibitors of CD73 over blocking antibodies. ATG-037 is being evaluated by Antengene in a Phase I trial as monotherapy and in combination with anti-PD-1 antibody in patients with locally advanced or metastatic solid tumors.

The novel ATR inhibitor ATG-018 is efficacious in preclinical cancer models

Abstract: 2604

Session: DNA Damage Response and Repair

Date and Time: 9:00 AM – 12:30 PM CST, April 12, 2022

Venue: Poster Section 22

In this study, the preclinical pharmacology data set supporting the development of ATG-018, a small molecule ATR inhibitor, was reviewed. Inhibiting ATR kinase (ataxia telangiectasia and Rad3 related kinase) leads to increased accumulation of double-strand breaks, particularly meaningful for tumor cells which rely on DNA damage response (DDR). ATG-018 was tested in a panel of 142 tumor cell lines and three CDX mouse models to assess anti-tumor efficacy and to identify potential predictive biomarkers. ATG-018 was a potent inhibitor of in vitro ATR activity inhibition and cell proliferation without significant impact on normal peripheral blood mononuclear cell (PBMCs) viability.

In addition, a series of genetic alterations were discovered that correlated with ATG-018 sensitivity and could be potential predictive biomarkers. As shown in Figure, the authors found that ATG-018 demonstrated potent in vivo efficacy in solid tumor/hematologic cancer models with certain DDR-related mutations. These data showed the potential of ATG-018 in synthetic lethality with homologous recombination deficiencies and promising application in a wide range of indications. With single-agent activity and no impact on PBMCs viability, ATG-018 may be well positioned for use in mono- or combination therapy in a wide range of tumors that rely on DDR. Development of a set of predictive biomarkers could enable its use as a precision-medicine. Antengene intends to file the first IND for ATG-018 in 2022.

ATG-022, an antibody-drug conjugate targeting Claudin 18.2, demonstrated potent in vivo efficacy in gastric cancer patient-derived xenografts

Abstract: 1143

Session: Preclinical and Clinical Pharmacology

Date and Time: 9:00 AM – 12:30 PM CST, April 11, 2022

Venue: Poster Section 25

In this preclinical study, ATG-022, an antibody-drug conjugate targeting Claudin18.2 (CLDN18.2), was evaluated in several gastric cancer patient-derived xenograft (PDX) models, to assess whether it had potential across a range of CLDN18.2 expression levels. Human CLDN18.2 is ectopically expressed in a large number of gastric and pancreatic cancers. Monoclonal antibody targeting CLDN18.2 demonstrated a promising clinical benefit when used in combination with chemotherapy. However, it showed suboptimal efficacy in patients with low CLDN18.2 levels.

In this study presented in the AACR (Free AACR Whitepaper), ATG-022 is reported to show high affinity (sub-nanomolar grade) against CLDN18.2 and demonstrated potent in vitro and in vivo antitumor effects, with in vivo efficacy observed in CLDN18.2 low expression PDX models. As shown in Figure, ATG-022 demonstrated much better in vivo efficacy compared with benchmark ADC. In addition, ATG-022 was highly specific for CLDN18.2, with virtually no effect on cells expressing CLDN18.1. In addition, ATG-022 has almost no impact on body weight, a proxy for safety. The authors concluded that ATG-022 shows promise for treating gastric cancer patients with a broad range of CLDN18.2 expression levels, a significant unmet need. Antengene is conducting preclinical studies for ATG-022.

Synergistic effects of the combination of Kras (G12C) with SHP2, ERK 1/2, mTORC1/2 or XPO1 inhibition for the treatment of Kras (G12C) mutated cancer

Abstract: 2679

Session: Signaling Pathway Inhibitors

Date and Time: 9:00 AM – 12:30 PM CST, April 12, 2022

Venue: Poster Section 25

This preclinical study was conducted to identify combination therapy regimen that could overcome the short progression free survival that is a characteristic of KRAS G12C inhibitors (linked to acquired resistance). The study evaluated the anti-tumor activity of ATG-012, a KRAS G12C inhibitor, with four other agents that are involved in the multiple pathways impacted by RASi: i) an SHP2 inhibitor (ET0038), ii) an ERK 1/2 kinase inhibitor (ATG-017), iii) an mTORC1/2 kinase inhibitor (ATG-008) or iv) the XPO-1 inhibitor, Selinexor, in preclinical solid tumor CDX models.

While ATG-012 monotherapy induced dose-dependent tumor growth inhibition at day 27, as shown in Figure, the authors also found strong in vivo synergism in 2-agent combinations. In particular, ATG-012 and clinical stage ERK inhibitor (ATG-017) demonstrate strong in vitro and in vivo synergism, suggesting potential clinical application which may overcome the rapid resistance of KRAS inhibitors. These data open the door to a range of combination partners for ATG-012 that could be fine-tuned to address drug resistance and potentially improve progression-free survival by matching tumor type/histology and combination partner for patients with the KRAS G12C mutation. Antengene is conducting preclinical studies for ATG-012.

Identification of MUC5B mutation as a positive predictive biomarker for mTORC1/2 inhibition by ATG-008 in lung cancer

Abstract: 4032

Session: Molecular Pharmacology

Date and Time: 9:00 AM – 12:30 PM CST, April 13, 2022

Venue: Poster Section 26

This study was designed to evaluate whether MUC5B could serve as a positive predictive biomarker for mTORC1/2 inhibition by ATG-008 (Onatasertib) in lung cancer. ATG-008 is a dual mTOR complex 1/2 kinase inhibitor. The mTOR complex regulates cell growth, metabolism, proliferation and survival. While the mTOR pathway is frequently deregulated in cancers, efficacy of mTOR inhibitors in lung cancer has been modest. In the study, 31 lung cancer cell lines were treated with ATG-008 to determine dose response and to correlate the gene mutation, amplification and expression with sensitivity to ATG-008.

As shown in Figure, the authors found that the presence of the MUC5B mutation correlates with more potent anti-tumor efficacy of ATG-008 in vitro and in vivo in lung cancer CDX models. The mucin MUC5B has a critical protective role in normal lung and has been identified as prognostic marker in multiple tumor types. One observation highlighted in the poster is that MUC5B is also mutated in melanoma, endometrial, colorectal, esophogastric and cervical cancers, vastly expanding the potential clinical utility of MUC5B mutation as the predictive biomarker for ATG-008. ATG-008 is being evaluated by Antengene in multiple Phase I and II clinical trials.

Gennao Bio Presents Promising New Results from its Gene Monoclonal Antibody (GMAB) Platform at the American Association for Cancer Research (AACR) 2022 Annual Meeting

On April 10, 2022 Gennao Bio, a privately held genetic medicines company developing first-in-class, targeted nucleic acid therapeutics, reported promising preclinical results of its proprietary, non-viral gene monoclonal antibody (GMAB) platform in multiple solid tumor models at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting (Press release, Gennao Bio, APR 10, 2022, View Source [SID1234611851]). The findings from these studies were reported and discussed in an oral presentation by Elias Quijano, M.D./Ph.D. candidate in the laboratory of Dr. Peter Glazer at the Yale School of Medicine, and co-founder of Gennao Bio, entitled, "Systemic targeting of therapeutic RNA to cancer via a novel, cell-penetrating and nucleic acid binding, monoclonal antibody."

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The preclinical results demonstrated GMAB’s ability to form non-covalent complexes with and systemically target and deliver 3p-hpRNA, a potent activator of the immune signaling RIG-I pathway, to solid tumors, including orthotopic mouse models of human pancreatic cancer (KPC) and medulloblastoma (DAOY). GMAB’s highly specific delivery into tumors is independent of the endocytic pathway and is uniquely enabled by targeting ENT2, a nucleoside transporter that is overexpressed in many tumors. In vitro studies of GMAB/3p-hpRNA demonstrated that delivery of a RIG-I agonist to tumor cells triggers an immune stimulating type-1 interferon response and triggers direct tumor cell death.

"These positive results further reinforce our strong belief in the broad therapeutic potential and diverse application of our GMAB platform in treating cancers with substantial unmet need," said Stephen Squinto, Ph.D., chief executive officer and chair of the board of Gennao Bio. "We expect to advance the humanized version of GMAB/3p-hpRNA, GMAB-7001, into Investigational New Drug (IND)-enabling studies in the second half of 2022 and will continue to assess additional oncology pipeline programs."

In the KPC pancreatic cancer model, multiple doses of GMAB/3p-hpRNA resulted in a significant survival benefit, driven in part by long-term increases in tumor-infiltrating lymphocytes, including CD45+, CD8+, CD4+, and CD19+ cells. GMAB/3p-hpRNA treatment also showed a statistically significant increase in tumor cell necrosis compared to the control group. Previous studies of a single dose administration of GMAB/3p-hpRNA in an orthotopic model of medulloblastoma demonstrated its ability to penetrate the central nervous system, reduce intracranial tumor burden by 50%, and prevent spinal metastases.

"The GMAB platform has the potential to address the challenges faced by alternative methods of delivery of immunostimulatory nucleic acids to tumors, which have been associated with systemic toxicities or rely on suboptimal intra-tumoral injections. Studies of single and multiple intravenous doses of GMAB/3p-hpRNA have shown targeted payload tumor delivery and resultant tumor growth suppression in several preclinical models of difficult-to-treat forms of cancer," said Mr. Quijano. "These promising monotherapy results, and the new data generated in a difficult-to-treat pancreatic cancer model, warrant continued research of the GMAB platform and development of this new class of targeted nucleic acid therapeutics for cancer."

A copy of the AACR (Free AACR Whitepaper) presentation can be found under the "News" section on the Company’s website, www.gennao.com.