On August 30, 2021 VBL Therapeutics (NASDAQ: VBLT) reported enrollment of new patients in VB-111 studies in the Unites States will resume immediately following authorization by the U.S. Food and Drug Administration (FDA) Chemistry, Manufacturing and Controls (CMC) Group to use new batches of ofranergene obadenovec (VB-111) produced in VBL’s commercial-scale GMP Modiin, Israel facility in clinical studies in the United States (Press release, VBL Therapeutics, AUG 30, 2021, View Source [SID1234587020]).

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In June, VBL was notified by the FDA that clearance of new VB-111 batches for clinical use in the United States was pending the completion of a technical review by the CMC group, which focused on the comparability of VB-111 manufacturing between different source sites. VBL prepared and submitted the requested data and documentation to the FDA in early August and the FDA has now provided clearance for VBL to use new batches of VB-111 produced in its commercial-scale facility located in Modiin, Israel. VBL has sufficient FDA-cleared batches and will resume patient recruitment in the OVAL trial in the United States.

The OVAL trial evaluating VB-111 in ovarian cancer is planned to enroll approximately 400 patients globally and nearly 80% of patients have already been recruited. The trial has two primary endpoints: progression free survival (PFS) and overall survival (OS). Successfully meeting either primary endpoint has the potential to support a biologics license application (BLA). Meeting the PFS endpoint, with a readout anticipated in the second half of 2022, could accelerate BLA submission by approximately one year, subject to discussions with the FDA, compared to original projections based on the readout of the OS primary endpoint that remains anticipated in 2023.

About VB-111 (ofranergene obadenovec; `ofra-vec`)
VB-111 is an investigational anti-cancer gene-therapy agent in development to treat a wide range of solid tumors. VB-111 is a unique biologic agent designed to use a dual mechanism to target solid tumors. Its mechanism combines the blockade of tumor vasculature with an anti-tumor immune response. VB-111 is administered as an IV infusion once every 6-8 weeks. It has been observed in past clinical research to be generally well-tolerated in >300 cancer patients and demonstrated activity signals in an "all comers" Phase 1 trial as well as in three tumor-specific Phase 2 studies. VB-111 has received orphan designation for the treatment of ovarian cancer by the European Commission. VB-111 has also received orphan drug designation in both the United States and Europe, and fast track designation in the United States, for prolongation of survival in patients with recurrent glioblastoma. VB-111 demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970).

About the OVAL Trial (NCT03398655)
OVAL (VB-111-701/GOG-3018) is an international Phase 3 randomized pivotal registration-enabling clinical trial comparing a combination of VB-111 and paclitaxel to placebo plus paclitaxel, in patients with platinum-resistant ovarian cancer. The trial is planned to enroll approximately 400 adult patients. OVAL is conducted in collaboration with the GOG Foundation, Inc., an independent international non-profit organization with the purpose of promoting excellence in the field of gynecologic malignancies.

On August 30, 2021 Illumina, Inc. (NASDAQ: ILMN) reported that its executives will be speaking at the following investor conference (Press release, Illumina, AUG 30, 2021, View Source [SID1234587019]):

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Morgan Stanley 19th Annual Global Healthcare Conference on September 13, 2021
Fireside Chat at 11:00 am Pacific Time (2:00 pm Eastern Time)
The live webcast can be accessed under the Investor Info section of the "company" tab at www.illumina.com. A replay will be posted on Illumina’s website after the event and will be available for at least 30 days following.

On August 30, 2021 InnoCare Pharma (HKEX: 09969), a commercial-stage biopharmaceutical company, reported the Investigational New Drug (IND) clearance of its second-generation pan-TRK inhibitor ICP-723 by the US Food and Drug Administration (FDA) for starting phase I clinical trial in the United States (Press release, InnoCare Pharma, AUG 30, 2021, View Source [SID1234587018]).

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Preclinical studies have shown that ICP-723 has a good safety profile and high activity against a variety of solid tumors, and the potential to provide a broad-spectrum anti-cancer therapy for patients with solid tumors with NTRK gene fusion. The clinical trial will evaluate the safety, tolerability and pharmacokinetic properties of ICP-723 in patients with solid tumors, and evaluate the anti-tumor efficacy of ICP-723 on NTRK fusion-positive cancers.

ICP-723 was developed to treat advanced or metastatic solid tumors harboring NTRK fusion genes, including breast cancer, colorectal cancer, lung cancer, thyroid cancer, etc., and for patients resistant to the first generation of TRK inhibitors.

Currently in China, ICP-723 is in the Phase I dose escalation (1mg, 2mg, 3mg and 4mg), ICP-723 showed efficacy in two patients with qualified neurotrophic tyrosine receptor kinase (NTRK) fusion. The NTRK fusion positive patient in 3 mg cohort reached stable disease (>20% tumor reduction) and the patient in 4 mg cohort achieved partial response at the first tumor assessment at the end of cycle 1, or day 28.

"I feel excited that ICP-723 becomes our third innovative drug entering the clinical stage in the United States," said Dr. Jasmine, the Co-founder, Chairwoman and CEO of InnoCare, "As a novel small molecule second generation pan-TRK inhibitor, ICP-723 has potent activity and high selectivity in preclinical experiments, which is expected to overcome the resistance to the first-generation TRK inhibitor to better benefit patients. "

On August 30, 2021 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for P-BCMA-ALLO1, the Company’s first fully allogeneic CAR-T product candidate for patients with relapsed/refractory multiple myeloma (Press release, Poseida Therapeutics, AUG 30, 2021, View Source;investigational-new-drug-application-for-p-bcma-allo1-an-allogeneic-car-t-candidate-for-relapsedrefractory-multiple-myeloma-301364920.html [SID1234587017]).

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"We view a fully allogeneic CAR-T product candidate comprised of a high-percentage of desirable stem cell memory T cells (Tscm) as the ‘holy grail’ of cell therapy in oncology," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida Therapeutics. "P-BCMA-ALLO1 has a very high percentage of Tscm cells with the potential to demonstrate safety in line with our prior P-BCMA-101 autologous approach, allowing for fully-outpatient dosing. The increase in Tscm and a switch to an improved binder also gives P-BCMA-ALLO1 the potential for even better efficacy."

Poseida announces FDA clearance of IND for its first fully #allogeneic CAR-T therapy for patients with multiple myeloma.

"Notably, Poseida’s propriety booster molecule technology gives us the ability to produce up to hundreds of doses of P-BCMA-ALLO1 from a single manufacturing run, thereby dramatically reducing cost and further increasing accessibility for patients who desperately need better and safer cell therapies," Ostertag continued.

With the P-BCMA-ALLO1 IND now cleared, the Company is actively focused on opening clinical sites with the intention to begin dosing later this year. P-BCMA-ALLO1-101 is a Phase 1 study comprised of open-label, dose escalation, multiple cohorts of allogeneic T stem cell memory (Tscm) CAR-T cells in subjects with relapsed/refractory multiple myeloma. This Phase 1 study follows a 3+3 design of dose-escalating cohorts. After a subject enrolls, allogeneic CAR-T cells will be administered as a single dose, following a standard chemotherapy-based conditioning regimen. Treated subjects will undergo serial measurements of safety, tolerability, and response. The study protocol allows for exploration of additional dosing regimens, including re-dosing, once initial safety has been established.

About P-BCMA-ALLO1
P-BCMA-ALLO1 is Poseida’s first fully allogeneic product candidate targeting B-cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloma. In in vitro and in vivo preclinical studies, P-BCMA-ALLO1 showed effective, targeted cancer cell killing and cytokine secretion, with similar or superior performance in anti-tumor efficacy compared to an autologous CAR-T therapy, P-BCMA-101. Inclusion of a proprietary "booster molecule" in the allogeneic manufacturing process further improved expansion of gene-edited cells and enabled production of hundreds of patient doses from a single manufacturing run, thereby reducing the manufacturing cost per dose into the same range as that of a monoclonal antibody.

On August 30, 2021 Lantern Pharma (NASDAQ: LTRN), a clinical stage biopharmaceutical company using its proprietary RADR artificial intelligence ("A.I.") platform to transform the cost, pace, and timeline of oncology drug discovery and development, reported that the U.S. Food and Drug Administration (FDA) has granted LP-184 Orphan Drug Designation (ODD) for the treatment of glioblastoma multiforme (GBM) and other malignant gliomas (Press release, Lantern Pharma, AUG 30, 2021, View Source;malignant-gliomas-301365111.html [SID1234587015]). This news follows the recent announcement of the FDA granting LP-184 ODD for the treatment of pancreatic cancer.

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LP-184 is a small molecule drug candidate and next generation alkylating agent that preferentially damages DNA in cancer cells that over-express certain biomarkers or that harbor mutations in DNA repair pathways. LP-184 is being developed for several targeted indications in cancer, including glioblastoma and pancreatic cancer.

GBM is a rare disease with an overall five-year survival rate of 5%. This means that only approximately 5 in 100 people survive GBM for five years and beyond. In 2020, 12,000 new GBM cases were diagnosed in the U.S. and more than 154,000 new cases were diagnosed worldwide. LP-184 acts by damaging DNA selectively in tumors that express high levels of the enzyme PTGR1. Analyses driven by RADR, Lantern’s proprietary machine learning-based artificial intelligence platform, have identified, in clinical databases, GBMs with elevated PTGR1 expression and harboring defects in DNA damage repair components as a targeted subset of genetically defined patients who could potentially benefit from LP-184-based therapy. According to market analysts at GlobalData, the global GBM market is expected to reach $1.8 billion USD in therapy sales and is growing at a CAGR of 12.8%.

"GBM represents an important, underserved clinical opportunity, with a significant unmet medical need," stated Panna Sharma, President & CEO of Lantern Pharma. "This second Orphan Drug Designation from the FDA for the LP-184 program marks another major milestone and is further validation of the power of our data-driven approach to oncology drug development, aimed at more targeted and effective oncology therapies."

"We recently reported positive preclinical data that demonstrated LP-184 inhibits tumor growth by greater than 106% and improved survival in animal models of GBM," continued Mr. Sharma. "This new data that we reported, in collaboration with the Kennedy Krieger Institute and Johns Hopkins, on the efficacy of LP-184 in GBM cell lines, in-vivo animal models, and in patient-derived neurospheres, validated in-silico predictions generated by our RADR A.I. platform. We believe LP-184’s ability to cross the blood-brain barrier, together with its anti-tumor efficacy and sensitivity correlations with relevant biomarkers, highlight LP-184’s potential to be used as both monotherapy as well as a synergistic agent in combination with other drugs to address the unmet needs in GBM and other aggressive central nervous system tumors."

"With our extended and expanded agreement with the Kennedy Krieger Institute and Johns Hopkins, we look forward to further advancing the potential of LP-184 as a new, potent treatment option for GBM, especially in areas of unmet clinical need, including MGMT-unmethylated, temozolomide (TMZ)-resistant GBMs, and also EGFR-aberrant or recurrent GBMs, all of which are often associated with poor prognosis and outcome for patients."

The FDA’s Office of Orphan Products Development grants orphan status to drugs intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation is designed to provide drug developers with various benefits to support the development of novel drugs, including market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, waiver of marketing registration application fees, reduced annual product fees, clinical protocol assistance and qualification for expedited development programs.