On July 9, 2020 Transgene (Paris:TNG) and Hypertrust Patient Data Care reported as a world premiere the first productive blockchain solution for clinical trials of personalized healthcare, leveraging the Hypertrust X-Chain for Clinical Trials product (Press release, Transgene, JUL 9, 2020, View Source [SID1234561804]). The cloud-based solution monitors and orchestrates the supply chain processes of Transgene’s AI-enhanced TG4050 individualized therapeutic vaccine based on the myvac technology, against ovarian, head and neck cancer providing an immutable audit trail throughout the treatment process.

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The innovative collaboration between Transgene and Hypertrust marks a milestone in the ongoing fight against cancer. The blockchain-based Hypertrust X-Chain solution supports Transgene’s personalized immunotherapies comprehensively, ensuring a trustworthy process in the background of the medical treatment. All emerging personalized treatments have a massive need for trustworthy and decentralized solutions, due to the strong cross-company collaborative nature of the production process.

Transgene’s first individualized immunotherapy product candidate is based on the myvac technology. It is currently evaluated in two ongoing clinical trials, including patients in Europe and in the USA. This virus-based therapeutic vaccine encodes neoantigens (patient-specific mutations) that are identified and selected using state-of-the-art artificial intelligence capabilities. myvac-based products are designed to stimulate the patient’s immune response, to recognize and destroy tumor cells based on their own mutations. This individualized immunotherapy is developed specifically for each patient.

"We have set up multiple collaborations around the world to allow each patient’s anticancer therapy to be designed in a timely manner. Blockchain technologies are a perfect tool to ensure that the patient’s genetic data are protected while ensuring that all the interventions of our partners are both smooth and tracked. We were very happy to collaborate with Hypertrust and together set up this novel process, demonstrating once again Transgene’s ability to be at the forefront of innovation", commented Eric Quéméneur, Pharm.D., Ph.D., Executive VP, Chief Scientific Officer of Transgene.

"We are very proud to present with our partner Transgene the world’s first productive blockchain solution for clinical trials of personalized healthcare. The combination of Transgene’s innovative and AI-enhanced TG4050 cancer vaccine, combined with the security and workflow orchestration features of our platform provides the optimum support for a highly personalized cancer treatment process", says Andreas Göbel, CEO of Hypertrust Patient Data Care.

Especially in clinical trials of personalized healthcare there is an essential need for an immutable audit trail, which is created by different parties throughout the supply and production process. Blockchain technologies bring a high level of trust, as it allows a total transparency on how the data were modified throughout the process and provide additional protection in an age of increasing cyberthreats.

Hypertrust X-Chain for Clinical Trials by Hypertrust Patient Data Care is a market-leading solution for the orchestration and documentation of clinical trials for personalized healthcare. It ensures the chain of identity and custody for personalized treatments in the context of clinical trials. The decentralized platform leverages blockchain technology, to provide closed-loop supply chain monitoring and orchestration, based on a workflow process definition. The solution provides an immutable and tamper-proof audit trail of all recorded data throughout the treatment. Also, it allows the migration towards the commercial scale solution "X-Chain for Commercialized Treatments".

On July 9, 2020 Indapta Therapeutics, Inc., a biotechnology company focused on developing and commercializing a proprietary, first-in-class, off-the-shelf, non-engineered, allogeneic FcRγ-deficient natural killer (G-NK) cell therapy to treat multiple cancers, reported an update on its progress in advancing the development of its programs (Press release, Indapta Therapeutics, JUL 9, 2020, View Source [SID1234561803]).

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"Since we emerged from stealth mode in January and announced our founding management team, scientific advisors and strategic partnership with Lonza, we have made substantial progress in advancing our next-generation, off-the-shelf, allogeneic immuno-oncology therapy," said Guy DiPierro, founder and chief executive officer of Indapta Therapeutics. "We have achieved several preclinical, regulatory and manufacturing milestones for our G-NK program, all of which are helping us move our novel program forward towards an IND submission and the initiation of a first-in-human trial in summer 2021."

Recent Program Updates

Presented data from its G-NK cell therapy program in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II in June. In the preclinical study, clinical models injected with human multiple myeloma cells were treated with a monoclonal antibody daratumumab or elotuzumab, which are FDA-approved for multiple myeloma, either alone or in combination with Indapta’s G-NK cells or comparator cells. In response, the G-NK cells demonstrated superior antibody-dependent cellular cytotoxicity (ADCC) and increased survival. G-NK cells show significantly enhanced expression of interferon-gamma (IFNγ) and tumor necrosis factor alpha (TNFα) compared with conventional natural killer (NK) cells.
Submitted a pre-Investigational New Drug (IND) briefing package to the U.S. Food and Drug Administration (FDA) for its G-NK cells. Indapta participated in a Type C meeting with the Agency and received written feedback outlining a clear and detailed roadmap for an IND application and for Chemistry, Manufacturing and Controls (CMC) activity.
Successfully completed the Stage 1 manufacturing transfer to Lonza, which conducted feasibility manufacturing runs at its Houston facility. Lonza will now initiate the next step of process development for the manufacturing of off-the-shelf, allogeneic G-NK cell therapy under current good manufacturing practices (cGMP) for use in the IND submission.
Indapta completed a persistence trial at the University of Houston which determined its G-NK cells remained in the body in both cryopreserved and frozen forms without any issues of functional exhaustion or phenotypic changes in vivo. Results demonstrated that Indapta’s cryopreserved G-NK cells remained in the body more than 26 times longer than cryopreserved conventional NK cells. Indapta also collected new data on cancer killing compounds including TNFα, IFNγ, CD107a, perforin and granzyme, all of which were expressed in higher amounts as a result of Indapta’s G-NK cell therapy.
Granted broad intellectual property protection through an exclusively-held patent related to manufacturing processes, G-NK cell selection and G-NK cell treatment used as a monotherapy and in combination with multiple compounds in multiple oncology and viral indications.
About Indapta’s G-NK Cell Therapy
Indapta Therapeutics is developing a universal, allogeneic G-NK cell therapy designed to substantially improve the cytotoxicity of monoclonal antibody therapy in multiple cancers. G-NK cells are a specific and potent subset of NK (natural killer) cells with specialized anti-tumor activity resulting from an epigenetic change, rather than engineering. Indapta has further enhanced G-NK cells via specific G-NK cell subset selection and its proprietary manufacturing process which, when combined, produce a G-NK cell therapy that demonstrates higher efficacy, persistence and enhanced cryopreservation.

When a monoclonal antibody binds to the tumor target and to Indapta’s G-NK cell therapy product, it initiates the release of dramatically more cancer killing compounds than conventional NK cells, allowing for increased efficacy and potentially less frequent dosing. Indapta’s off-the-shelf G-NK cell therapy is further differentiated from other NK cell therapies in that it is a cell banked product with low variability. In vivo studies have demonstrated the safety of Indapta’s G-NK cell therapy.

On July 9, 2020 Eagle Pharmaceuticals, Inc. ("Eagle" or the "Company") (NASDAQ: EGRX) reported that the Centers for Medicare & Medicaid Services (CMS) has established a unique, product-specific billing code for PEMFEXY (pemetrexed for injection) (Press release, Eagle Pharmaceuticals, JUL 9, 2020, View Source [SID1234561802]). This new Healthcare Common Procedure Coding System (HCPCS) code, or J-code, is J9304 (Injection, pemetrexed (PEMFEXY), 10 mg). The J-code will become effective on October 1, 2020.

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The new HCPCS code provides coding clarity to outpatient facilities and physicians who will administer PEMFEXY, facilitating access for patients and reimbursement from Medicare, Medicaid and commercial insurance.

In February 2020, Eagle received final approval of its New Drug Application (NDA) from FDA for PEMFEXY, a branded alternative to ALIMTA, following settlement of patent litigation with Eli Lilly and Company. The Company is entitled to initial market entry (equivalent to approximately a three-week supply of current ALIMTA utilization) on February 1, 2022, and a subsequent uncapped entry on April 1, 2022.

The Company also received a supplement approval from FDA for a 500mg multiple-dose vial of PEMFEXY on June 18, 2020.

About PEMFEXY

PEMFEXY is a pemetrexed injection ready-to-dilute formulation for locally advanced or metastatic nonsquamous non-small cell lung cancer in combination with cisplatin; locally advanced or metastatic nonsquamous non-small cell lung cancer whose disease has not progressed after four cycles of platinum-based first-line chemotherapy, as maintenance treatment; locally advanced or metastatic nonsquamous non-small cell lung cancer after prior chemotherapy as a single agent; and malignant pleural mesothelioma whose disease is unresectable or who are otherwise not candidates for curative surgery in combination with cisplatin.

On July 9, 2020 JS InnoPharm Ltd reported the enrollment of the first patient in a Phase 1, open-label, monotherapy dose-escalation and expansion study of JSI-1187, the company’s lead investigational drug candidate targeting the enzymes ERK1 and ERK2 (Press release, JS InnoPharm, JUL 9, 2020, View Source [SID1234561801]). The study is being conducted at 4 NCI-designated Comprehensive Cancer Centers in the U.S. JS InnoPharm holds the U.S. IND for JSI-1187, and this trial is being managed by Strategia Pharmaceuticals LLC, JS InnoPharm’s clinical development partner in the United States. Initiation of this clinical trial represents an important step in the company’s efforts to discover and develop precision medicine for cancer patients.

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The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical activity of JSI-1187 in patients with relapsed, refractory solid tumors with MAPK mutations. The study will comprise three components: (1) a dose escalation phase of JSI-1187 monotherapy for patients with solid tumors, (2) a dose escalation phase of JSI-1187 in combination with BRAF inhibitor, dabrafenib, and (3) an expansion phase for patients with tumors harboring specific mutations.

"JSI-1187 is a selective ERK1/2 inhibitor. By utilizing an ERK inhibitor against tumors harboring mutations in the MAPK pathway, we hope to develop more effective single-agent and combination therapies by preventing the early development of resistance seen with current MAPK targeted agents," said Dr. Linda Paradiso, Chief Development Officer for Strategia Pharmaceuticals.

The Role of ERK in Cancer

MAPK signaling via the RAS-RAF-MEK-ERK cascade plays a critical role in cancer growth and proliferation. Alterations in the MAPK/ERK pathway are found in a variety of cancer types, with KRAS mutations in pancreatic (>90%), biliary tract (3-50%), colorectal (30-50%), lung (25-30%), ovarian (15-39%) and endometrial (18%) cancers; NRAS mutations in 20% of melanoma; and BRAF V600 mutations in 50% of melanoma and 7% of a variety of other tumor types. Although it has been demonstrated that BRAF/MEK inhibitor-targeted combination therapy provides a significant benefit beyond single agent therapy in melanoma and other cancers, the majority of patients eventually develop resistance and disease progression. Several mechanisms of acquired resistance have been identified and central to these mechanisms is the reactivation of ERK signaling. Therefore, ERK inhibition may provide the opportunity to avoid or overcome resistance from upstream mechanisms, as it is the most distal kinase of this signaling pathway.

About JSI-1187

JSI-1187 is a selective and orally administered small molecule inhibitor of ERK1 and ERK2. In preclinical studies, it demonstrated high potency against a variety of tumors with MAPK pathway mutations.

On July 9, 2020 Kronos Bio, Inc., dedicated to targeting oncogenic transcription factors and their associated transcriptional regulatory networks, reported results of a preclinical study of KB-0742, a highly potent, orally available and selective cyclin-dependent kinase 9 (CDK9) inhibitor. Results showed that KB-0742 inhibited tumor growth in a prostate xenograft model, as well as other cancers addicted to high levels of oncogenic transcription. The findings were presented in a poster session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II in June 2020 (Press release, Kronos Bio, JUL 9, 2020, View Source [SID1234561800]).

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"Current therapies that block androgen signaling are effective in prostate cancer, but most ultimately develop resistance. These preclinical study results provide strong evidence that KB-0742 inhibits tumor growth in vivo," said Norbert Bischofberger, Ph.D., President and Chief Executive Officer of Kronos. "Based in part on these data, we plan to advance KB-0742 into clinical development for the treatment of transcriptionally-addicted cancers. We anticipate submitting an IND in the fourth quarter of this year and initiating a Phase 1 dose-escalation study in early 2021."

In this study, which was conducted in collaboration with the Koehler Lab at the MIT Center for Precision Cancer Medicine and Koch Institute For Integrative Cancer Research at MIT, researchers used the small molecule microarray (SMM) discovery platform to identify novel modulators of the androgen receptor capable of overcoming therapy resistance in prostate cancer cells. The SMM screen identified KI-ARv-03, a small molecule that blocks androgen receptor dependent gene expression and is a highly selective inhibitor of the androgen receptor cofactor CDK9. KB-0742 is a more potent and drug-like small molecule CDK9 inhibitor designed and optimized at Kronos Bio.

KB-0742 showed selectivity for CDK9 over other CDK family members, downregulated AR-dependent oncogenic transcription, and reduced tumor cell growth and promoted apoptosis in vitro. Additionally, oral administration of KB-0742 (administered as a 3-day on/4-day off regimen) to mice that had been engrafted with castration resistant prostate cancer cells significantly inhibited tumor growth with modest effects on body weight. In a subsequent mouse xenograft study using a MYC-dependent acute myeloid leukemia model, KB-0742 administration again resulted in significant tumor growth inhibition with dose-dependent effects on pharmacodynamic markers of CDK9 inhibition in tumor.