On June 27, 2019 Advaxis, Inc. (NASDAQ:ADXS), a clinical-stage biotechnology company focused on the discovery, development and commercialization of immunotherapy products, reported that it is increasing its focus on neoantigen-directed immunotherapies and closing the AIM2CERV Phase 3 clinical trial with axalimogene filolisbac (AXAL) in high-risk locally advanced cervical cancer (Press release, Advaxis, JUN 27, 2019, View Source [SID1234537303]). Advaxis intends to continue to support the clinical development of AXAL, its single-antigen construct, in other HPV-related cancers while redirecting resources towards advancing its neoantigen-directed programs. Specifically, the company plans to continue developing ADXS-NEO, currently in a Phase 1 clinical trial, in patients with several types of advanced or metastatic solid tumors including melanoma, lung, colorectal, head and neck and bladder cancers, and ADXS-HOT, currently in a Phase 1/2 clinical trial, for non-small cell lung cancer. The company anticipates advancing additional drug constructs from its ADXS-HOT program into the clinic over the next 18 months.

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"We remain firmly committed to our Lm Technology platform, including our personalized and off-the-shelf approaches for neoantigen-directed therapies, and the development of AXAL against HPV-related cancers," said Kenneth A. Berlin, President and Chief Executive Officer of Advaxis. "While closing the AIM2CERV trial was a difficult decision due to the efforts of many individuals including the investigators and patients, the delays we incurred as a result of the recent FDA partial clinical hold and the estimated cost and time to completion for AIM2CERV led us to believe the best path forward for the company is to focus on our neoantigen programs. We believe this increased focus will enable a quicker and more cost-effective approach to demonstrate the strength and versatility of our Lm platform, thereby enhancing shareholder value."

The estimated remaining cost to complete the AIM2CERV trial ranges from $80 million to $90 million, and initial efficacy data is not anticipated for at least three years. Therefore, results from the clinical trial were not the basis for the decision to close the study, nor was safety as the trial recently underwent its third Independent Data Monitoring Committee (IDMC) review with no safety issues noted. The company plans to unblind the AIM2CERV clinical data generated to date and anticipates submitting these data for publication. In addition, Advaxis will continue to pursue monetization opportunities for AXAL.

"The emerging data from our neoantigen programs look very promising, and therefore these programs merit an increased focus of the company’s resources," stated Mr. Berlin. "With this redirection of resources to our neoantigen programs, we anticipate our cash usage for the next 12 months will be in the range of $33 million to $37 million, which includes $8 million to $9 million in non-recurring costs associated with prior AXAL studies including AIM2CERV. This reduction in our cash burn is a significant improvement over the past several years and also betters the goal we set of $45 million at the beginning of our fiscal year 2019." He concluded, "We believe these changes will enable us to pursue a leadership position in the neoantigen field by building upon the early and exciting data from our ADXS-NEO and ADXS-HOT programs."

On June 27, 2019 Propanc Biopharma, Inc. (OTC: PPCBD) ("Propanc"), a biopharmaceutical company developing new cancer treatments for patients suffering from recurring and metastatic cancer, reported that Propanc’s intellectual property ("IP") portfolio has undergone rapid growth recently, with sixty-five patents currently either in force or pending in most major countries and regions around the world (Press release, Propanc, JUN 27, 2019, View Source [SID1234537304]). The IP covers Propanc’s underlying anti-cancer technology in development. In the past year, three additional patent families entered national phase, where a patent application is filed in individual countries and regions, in order to achieve grant status.

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"Our IP portfolio is a world leader in the field of administration of proenzymes for the treatment and prevention of metastatic cancer. IP is the cornerstone of any biotech company, and places us in a strong position to unlock significant value, as we plan to progress into a First-In-Human study and then hopefully towards proof of concept in a specific cancer indication, where we will then look at possible licensing opportunities," said Dr. Julian Kenyon, Propanc’s Chief Scientific Officer. "We are firmly of the opinion that our current market value does not truly reflect the advancement of our lead product, PRP, or its true potential as a breakthrough, long term treatment for aggressive and fast spreading cancers from solid tumors by targeting and eradicating cancer stem cells."

Propanc has a total of four patent families covering the use of proenzymes for treating cancer via a combination of trypsinogen and/or chymotrypsinogen pancreatic proenzymes, which includes Propanc’s lead product candidate, PRP. The IP portfolio includes composition of matter claims and a method of treatment to eradicate cancer stem cells. Further applications are also expected relating to the formulation and methods of use, as well as research programs with its joint research partners designed to further optimize the quality, safety and performance of PRP.

On June 26, 2019 Precigen, Inc., a wholly-owned subsidiary of Intrexon Corporation (NASDAQ: XON) and a biopharmaceutical company specializing in the development of innovative gene and cellular therapies to improve the lives of patients, reported that the first patient has been dosed with PRGN-3006, a first-in-class investigational therapy using Precigen’s non-viral UltraCAR-T therapeutic platform (Press release, Intrexon, JUN 26, 2019, View Source [SID1234537270]). PRGN-3006 UltraCAR-T is an autologous chimeric antigen receptor T (CAR-T) cell therapy under investigation for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndrome (MDS) (clinical trial identifier: NCT03927261).

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PRGN-3006 utilizes Precigen’s transformative UltraCAR-T therapeutic platform, which eliminates ex vivo expansion and reduces manufacturing time to fewer than two days following non-viral gene transfer at the cancer center. PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment utilizing Precigen’s advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control in targeting relapsed or refractory AML and higher risk MDS. The study is conducted in collaboration with the Moffitt Cancer Center.

"The first patient dosed using Precigen’s UltraCAR-T therapeutic platform is an important milestone for our company," said Helen Sabzevari, PhD, President of Precigen. "Timing is critical for this patient population and the ability to manufacture PRGN-3006 UltraCAR-T overnight without ex vivo expansion accelerates timing to provide treatment to patients."

"AML is an aggressive disease with very poor prognosis," said James J. Mulé, PhD, Associate Center Director and Michael McGillicuddy, Endowed Chair in Melanoma Research/Treatment at the Moffitt Cancer Center. "The first patient dosed with investigational PRGN-3006 UltraCAR-T represents a significant development for this challenging patient population with high unmet medical need."

The PRGN-3006 UltraCAR-T clinical study is a single center, nonrandomized, investigator‐initiated Phase 1/1b safety and tolerability study. The safety and tolerability of PRGN‐3006 UltraCAR-T will be assessed following intravenous administration of escalating doses in patients with relapsed or refractory AML or higher risk MDS.

"AML and MDS patients have few treatment options, and time is critical when selecting the best treatment path," said David A. Sallman, MD, lead investigator for the PRGN-3006 study at the Moffitt Cancer Center. "We are hopeful that this study will be the beginning of the development of a therapeutic that may result in a critically needed new safe and efficacious treatment option that allows for rapid treatment."

About Acute Myeloid Leukemia (AML)
AML is a cancer that starts in the bone marrow, but most often moves into the blood1. Though considered rare, AML is among the most common types of leukemia in adults2. In 2019, it is estimated that 21,450 new cases of AML will be diagnosed in the US2. AML is uncommon before the age of 45, and the average age of diagnosis is about 682. The prognosis for patients with AML is poor with an average 5‐year survival rate of approximately 25 percent overall and less than a 5 percent 5‐year survival rate for patients older than 653. Amongst elderly AML patients (≥ 65 years of age) median survival is short, ranging from 3.5 months for patients 65 to 74 years of age to 1.4 months for patients ≥ 85 years of age3.

About Myelodysplastic Syndromes (MDS)
MDS are cancerous conditions of the bone marrow4 generally found in adults in their 70s5. Incidence in the US is not known for sure, but estimates range from 10,000 each year and higher5. Sometimes referred to as pre-leukemia, about 1 in 3 MDS patients progress to AML4. As diseases of the bone marrow, MDS outlook is not based on the size of a tumor or tumor metastasis, and other factors are used to predict outlook and inform the treatment plan. Some factors have been combined to develop scoring systems, such as the Revised International Prognostic Scoring System (IPSS-R)6. Using the IPSS-R6, median survival for MDS patients can be estimated to vary from less than one year for the "very high" IPSS-R risk group to more than eight years for the "very low" IPSS-R group7.

Precigen : Advancing Medicine with PrecisionTM
Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cellular therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Precigen also follows the science opportunistically in pursuit of promising programs in emerging therapeutics. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. Precigen was founded as a wholly-owned subsidiary of Intrexon Corporation (NASDAQ: XON) and leverages a diverse portfolio of technology platforms to advance human health. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

Precigen’s UltraCAR-TTM Therapeutic Platform
Precigen’s UltraCAR-T platform has the potential to disrupt the CAR-T treatment landscape by increasing patient access through shortening manufacturing time, decreasing manufacturing-related costs, and improving outcomes using advanced approaches for precise tumor targeting and control of the immune system. The platform brings several key advancements: 1) Non-viral gene transfer using multigenic vectors for expression of multiple effector genes leads to better precision and control of tumor targeting and eliminates the need for virus; 2) Sustained persistence and desired phenotype of infused UltraCAR-T helps address T-cell exhaustion, a common issue with current CAR-T therapies; 3) T-cell control by incorporation of kill switch technology to potentially improve the safety profile; and 4) Rapid manufacturing of UltraCAR-T cells using our proprietary non-viral gene transfer process, which eliminates the need for ex vivo propagation, thus dramatically reducing wait times for patients from weeks to fewer than two days.

On June 26, 2019 Tikcro Technologies Ltd. (OTCQB: TIKRF), a pre-clinical stage developer of antibodies for cancer immune-therapy, reported its financial results for the first quarter ended March 31, 2019 (Press release, Tikcro, JUN 26, 2019, View Source [SID1234537286]).

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"We are focused on the progress of our cytotoxic T lymphocyte-associated antigen 4 (CTLA-4) antibody, which has shown strong comparative results in pre-clinical cancer treatment assays," said Aviv Boim, CEO of Tikcro. "At recent industry events a number of biotechnology companies and clinical centers have reported results of trial for the combination treatment of PD1 or PD-L1 antibodies with a CTLA-4 antibody. Several molecule structures and a number of experimental clinical indications in oncology were presented. These development efforts aim to broaden clinical indications, increase the efficacy and reduce immune-related adverse effects of a CTLA-4 antibody treatment. CTLA-4 antibody treatment gains additional attention, however, along with increased competition. Based on pre-clinical results, our novel CTLA-4 antibody has the potential to address the needs to offer higher efficacy and lower side effects."

Several established and emerging pharma companies, including Tikcro, are pursuing new CTLA-4 antibodies to further broaden its clinical scope and to reduce immune related adverse effects.

Financial Results for the First Quarter Ended March 31, 2019
Net loss for the first quarter of 2019 was $271,000, or $0.03 per diluted share, compared to a net loss of $377,000, or $0.04 per diluted share, for the same period last year.

As of March 31, 2019, the company reported $4.95 million in cash, cash equivalents and short-term bank deposits.

On June 26, 2019 TARIS Bio, a biopharmaceutical company developing transformational therapies to treat people with debilitating urological disorders, reported positive safety, tolerability and preliminary efficacy data from the full patient cohort (n=20) of its Phase 1b study of TAR-200 for the treatment of patients with muscle invasive bladder cancer (MIBC), neoadjuvant to radical cystectomy (RC) (Press release, TARIS Biomedical, JUN 26, 2019, View Source [SID1234537288]). This complete dataset builds on the initial positive signal observed in the first part of the study, announced previously. Combined data from both TAR-200 study arms indicates a robust antitumor response on final histopathologic analysis after cystectomy, favorable tolerability profile, and no detectable systemic drug exposure or associated adverse events. These data provide early evidence of the potential benefit of TAR-200 in the treatment of MIBC for both patients who can receive RC and those who are unfit for surgical intervention.

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"The TAR-200-101 study has revealed exciting results in patients with muscle invasive bladder cancer," said Siamak Daneshmand, M.D., Director of Urologic Oncology at the USC Institute of Urology, and principal investigator of the study. "Such evidence of response, while early, may enable definitive treatment in patients currently unfit for curative intent therapy, including treatment with checkpoint inhibitors. Moreover, TAR-200 may also ultimately enable organ preservation strategies in lieu of radical cystectomy in patients who are unfit or unwilling to undergo bladder removal."

Each arm in the multi-center study enrolled 10 subjects with organ-confined MIBC (defined as clinical T2-T3 N0M0 disease), who were scheduled to undergo RC and were deemed unfit for systemic platinum-based neoadjuvant chemotherapy. During the window between diagnosis and RC, the TAR-200 system, which continuously delivers gemcitabine in the bladder, was administered twice for seven days per dose separated by a two-week rest. There was no delay to RC for any patient in either arm, with surgery proceeding as planned on Day 28 in Arm 1 and Day 42 in Arm 2. Pathological assessment of pooled data from 20 subjects across the two arms demonstrates:

A 50% objective response rate (ORR), defined as no residual muscle invasive disease (greater than or equal to pT2 disease on post-surgical pathology)
A 40% complete response rate (CR), defined as only residual pT0 or pTis disease
No detectable systemic gemcitabine exposure, nor any typical gemcitabine-associated systemic adverse events
Excellent local tolerability and no evidence of dose-limiting toxicity
"These data provide strong, histopathologically confirmed evidence of TAR-200’s anti-tumor activity and highlight our product candidate’s potential to fundamentally change the management of the full spectrum of disease for patients with MIBC," said Tony Kingsley, President and CEO of TARIS. "The study results support our next stage of development that focuses first on the large portion of patients who do not receive potentially curative therapies. These patients typically have significant comorbidities and a poor overall performance status, rendering them unfit for current standards of care. With few options, these patients face rapid mortality from this unmanaged disease. We believe that TAR-200 is uniquely positioned to address this substantial and serious unmet need."

The standard of care for treatment of MIBC includes radical cystectomy (complete removal of the bladder and surrounding pelvic organs), with or without neoadjuvant chemotherapy. Radical cystectomy is a major, life-changing surgery, and many patients are medically unfit and/or unwilling to undergo this procedure. Without treatment, patients with MIBC who are not candidates for curative intent therapy quickly succumb to their disease.

TARIS is currently conducting a Phase 1b trial of TAR-200 in patients with MIBC who are ineligible for radical cystectomy, to evaluate the safety and tolerability of the system following four consecutive 21-day dosing cycles (TAR-200-103). The multicenter study rapidly over-enrolled 35 subjects – over three times the targeted number of subjects, demonstrating very high demand for a novel therapeutic option for this patient population. TARIS expects to report topline results from this study in late 2019. More information is available at www.clinicaltrials.gov, identifier # NCT03404791.

About Bladder Cancer
Bladder cancer is the fifth most common neoplasm in industrialized countries, affecting roughly 2.7 million people worldwide. The National Cancer Institute estimates approximately 81,200 new cases and 17,240 deaths due to this disease in 2018; muscle invasive bladder cancer (MIBC) accounts for 20-25% of the newly diagnosed cases and the majority of disease-related mortality. Approximately 8,500 of these patients are unfit for radical bladder removal or chemotherapy, and the only treatment option is palliative care.

About TAR-200
TARIS’ lead investigational product, TAR-200, is designed to release the chemotherapeutic agent gemcitabine continuously in the bladder for multiple weeks. TARIS believes that TAR-200 has the potential to harness gemcitabine’s both direct antitumor and immuno-oncologic activity without meaningful systemic drug exposure, yielding a powerful therapy and mitigating systemic side-effects. The TARIS System has been administered in clinical settings to over 450 individuals and over 900 doses across multiple urologic diseases. The U.S. Food and Drug Administration has granted Fast Track designation to TAR-200 in patients with organ-confined or locally advanced MIBC who are unfit for curative-intent therapy.