On December 14, 2017 OncoSec Medical Incorporated ("OncoSec" or the "Company") (NASDAQ:ONCS), a company developing intratumoral cancer immunotherapies, reported the initiation of patient dosing in PISCES/KEYNOTE-695, the company’s global, multi-center, registration-directed open-label Phase 2b clinical trial (Press release, OncoSec Medical, DEC 14, 2017, View Source [SID1234522642]). The trial will evaluate the combination of ImmunoPulse IL-12 (intratumoral pIL-12 [tavokinogene telseplasmid or "tavo"] with electroporation), and pembrolizumab in patients with unresectable metastatic melanoma who have progressed or are progressing on an anti-PD-1 therapy.

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"There remains a significant unmet medical need in oncology for patients in whom the existing PD-1 therapies do not work. Based on the encouraging data we presented at SITC (Free SITC Whitepaper), we believe the combination of ImmunoPulse IL-12 and pembrolizumab may offer a differentiated approach to reshaping the tumor microenvironment by converting immunologically cold tumors to hot," said Sharron Gargosky, Chief Clinical and Regulatory Officer of OncoSec. "We look forward to the continued advancement of this trial and to sharing data in 2018."

The Phase 2b, multicenter study of intratumoral tavo with electroporation in combination with intravenous pembrolizumab will enroll approximately 48 patients with a histological diagnosis of melanoma with progressive locally advanced or metastatic disease defined as Stage III or Stage IV. The primary endpoint will be the Best Overall Response Rate (BORR).

The Company’s prior Phase 2 OMS I-102 combination study of ImmunoPulse IL-12 and pembrolizumab in 22 patients unlikely to respond to anti-PD-1 therapy demonstrated a 50% best overall response rate and a 41% complete response rate. In addition, the trial showed a 57% progression free survival (PFS) rate at 15 months (median PFS not yet reached) and 100% (11/11) duration of response. In clinical studies to date, intratumoral tavo has demonstrated a favorable safety profile and has been well tolerated.

PISCES/KEYNOTE-695 is the second combination study conducted with pembrolizumab and, if successful, could form the basis for a BLA under the accelerated approval pathway.

To learn more about the trial, visit www.oncosec.com. Additional details can also be found at www.clinicaltrials.gov via NCT03132675.

About Metastatic Melanoma

Melanoma is a type of skin cancer that begins in skin cells called melanocytes. As the cancer progresses, melanoma becomes more difficult to treat once it spreads beyond the skin, such as the lymphatic system (metastatic disease). Given its occurrence in young individuals, the potential years of life lost to melanoma can be higher when compared with other cancers. Although melanoma is a rare form of skin cancer, it accounts for over 75% of skin cancer deaths. The American Cancer Society estimates that approximately 87,000 new melanoma cases and 10,000 deaths from the disease will occur in the United States in 2017. Additionally, the World Health Organization estimates that approximately 132,000 new cases of melanoma are diagnosed around the world every year.

About PISCES (Anti-PD-1 IL-12 Stage III/IV Combination Electroporation Study)/KEYNOTE-695

PISCES/KEYNOTE-695 is a global, multicenter phase 2b, open-label trial of intratumoral plasma encoded IL-12 (tavokinogene telseplasmid or "tavo") delivered by electroporation in combination with intravenous pembrolizumab in patients with stage III/IV melanoma who have progressed or are progressing on either pembrolizumab or nivolumab treatment. The Simon 2-stage study of intratumoral tavo plus electroporation in combination with pembrolizumab will enroll approximately 48 patients with histological diagnosis of melanoma with progressive locally advanced or metastatic disease defined as Stage III or Stage IV. The primary endpoint will be the Best Overall Response Rate (BORR).

To learn more about the trial, visit www.oncosec.com. Additional details can also be found at www.clinicaltrials.gov via NCT03132675.

PharmaCyte Biotech has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, PharmaCyte Biotech, 2017, DEC 14, 2017, View Source [SID1234522648]).

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On December 14, 2017 Aura Biosciences, a biotechnology company developing a new class of therapies to target and selectively destroy cancer cells using viral nanoparticle conjugates, reported that researchers at Massachusetts Eye and Ear (MEE), Emory University and the National Cancer Institute (NCI) have generated preclinical data that demonstrate the ability of light-activated AU-011 to target and selectively destroy ocular melanoma tumors, both in vitro and in vivo, in an orthotopic animal model that highly mimics the location and progression of the disease in humans(Press release, Aura Biosciences, DEC 14, 2017, View Source [SID1234522650]). These results have been published in the peer-reviewed journal, Molecular Cancer Therapeutics.

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Aura’s lead program, light-activated AU-011, is being developed for the treatment of primary ocular melanoma, a rare and life-threatening disease with no approved FDA therapies, and is currently in Phase 1b/2 clinical testing. Researchers believe AU-011’s high tumor specificity and targeting capability, due to its ability to selectively bind to heparan sulfate proteoglycans (HSPG) on the surface membrane of tumor cells, may be key to avoiding damage to the main ocular structures and preserving patients’ vision

"These data offer strong support for the basis of our clinical program, which we are currently investigating in patients with primary ocular melanoma," said Elisabet de los Pinos, Ph.D., founder and CEO of Aura. "Our ultimate goal is to offer a first-line treatment option that can achieve local tumor control, while enabling patients to maintain their vision."

Currently available treatment options to control local tumor growth, such as plaque radiotherapy, are highly invasive and not tumor-specific, often resulting in severe and vision-threatening complications. The data published today suggest that AU-011 may be further investigated as a novel first-line treatment option that can enable early intervention and potentially become the standard of care for patients with early-stage ocular melanoma.

About ocular melanoma
Ocular melanoma, also known as uveal or choroidal melanoma, is a rare and aggressive eye cancer. Ocular melanoma is the most common primary ocular tumor and develops in the uveal tract of the eye. No targeted therapies are available at present, and current radiotherapy treatments can be associated with severe visual loss and other long-term sequelae such as dry eye, glaucoma, cataracts and radiation retinopathy. The most common current treatment is plaque radiotherapy, which involves surgical placement of a radiation device against the exterior of the eye over the tumor. This technique can control the melanoma but can also lead to radiation-related cataract, retinopathy, optic nerve damage and loss of vision. The alternative is enucleation, or removal of the eye. Ocular melanoma metastasizes to the liver in about 40 percent of cases in the long-term (source: OMF), and only 15 percent of patients whose melanoma has metastasized survive beyond five years after diagnosis (source: ACS).

About light-activated AU-011
AU-011 is a first-in-class targeted therapy in development for the primary treatment of ocular melanoma. The therapy consists of viral nanoparticle conjugates that bind selectively to unique receptors on cancer cells in the eye and is derived from technology originally pioneered by Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI), recipient of the 2017 Lasker-DeBakey Award. Upon activation with an ophthalmic laser, the drug rapidly and specifically disrupts the membranes of tumor cells while sparing key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of treatment. This therapy can be delivered using equipment commonly found in the ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for ocular melanoma has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration and is currently in clinical development.

On December 14, 2017 Jnana Therapeutics Inc. reported a $50 million Series A financing (Press release, Jnana Therapeutics, DEC 14, 2017, View Source [SID1234529783]). The company is building the first drug discovery platform dedicated to targeting the solute carrier (SLC) family of transporters, the cell’s metabolic gates. Jnana’s proprietary small molecule platform allows the company to address therapeutic targets rapidly and comprehensively across the SLC transporter family.

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"SLC transporters provide cells with knowledge about their environment. These proteins are amongst the oldest in biology, and afford avenues to address virtually all major diseases. After 50 years of drug development, only a handful of SLCs have been harnessed to treat disease, leaving over 400 members unstudied. Our proprietary chemistry and biology platform allows us to systematically advance medicines based on the biology of these ancient information gathering systems," said Amir Nashat, Ph.D., Managing Partner with Polaris Partners and Chief Executive at Jnana. "We are targeting a broad range of medical conditions with significant unmet need, and we are fortunate to have the support of leaders in medicine and biotechnology."

Jnana is focusing on immunometabolism, lysosomal function and mucosal defense, important disease pathways where SLC transporters provide novel targets for immuno-oncology, inflammatory disorders and neurological diseases.

Seed funders Polaris Partners and Avalon Ventures were joined in the Series A syndicate by Versant Ventures, AbbVie Ventures, and Pfizer R&D Innovate.

Jnana’s founding team includes recognized leaders at the forefront of chemistry, drug discovery and development, translational medicine and company building. The company’s founders are:

Stuart Schreiber, Ph.D., Morris Loeb Professor at Harvard University, Howard Hughes Medical Institute Investigator and Cofounder of Broad Institute and biotech companies including Vertex, Ariad and H3 Biomedicine.
Ramnik Xavier, M.D., Ph.D., Chief of Gastroenterology at Massachusetts General Hospital, Professor at Harvard Medical School and Institute Member at Broad Institute.
Joanne Kotz, Ph.D., President at Jnana, previously Director at Broad Institute.
Joel Barrish, Ph.D., Chief Scientific Officer at Jnana and former VP and Head of Discovery Chemistry at Bristol-Myers Squibb.
Dr. Nashat of Polaris Partners will be joined on Jnana’s board of directors by Kevin Kinsella, founder of Avalon Ventures, and Carlo Rizzuto, Ph.D. Partner at Versant.

On December 14, 2017 Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the company has filed an Investigational New Drug (IND) application with the Division of Oncology at the U.S. Food and Drug Administration (FDA) for a proposed Phase 1 study of CLR 131 in children and adolescents with select rare and orphan designated cancers (Press release, Cellectar Biosciences, DEC 14, 2017, View Source [SID1234522651]).

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The proposed Phase 1 clinical trial of CLR 131 is an open-label, sequential-group, dose-escalation study to evaluate the safety and tolerability of a single intravenous administration of CLR 131 in up to 30 children and adolescents with cancers including neuroblastoma, sarcomas, lymphomas (including Hodgkin’s lymphoma) and malignant brain tumors. Secondary objectives of the study are to identify the recommended Phase 2 dose of CLR 131 and to determine preliminary antitumor activity (treatment response) of CLR 131 in children and adolescents.

The study will be initiated with the pediatric oncologists and Nuclear Medicine/Radiology Group at The University of Wisconsin Carbone Cancer Center.

"The University of Wisconsin group makes an ideal partner for the development of CLR 131 in pediatric cancers because of the quality of their investigators, prominence as a leading U.S. pediatric treatment center and extensive experience with beta-emitting radioisotope therapies. Together, our hope is to bring new and effective treatment options for children battling life-threatening cancers," stated John Friend, M.D., chief medical officer of Cellectar Biosciences.

CLR 131 is an investigational phospholipid drug conjugate (PDC), radioiodinated cancer therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ethers (PLEs) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues.

Dr. Otto and co-workers of The University of Wisconsin have demonstrated uptake of CLR 131 and other fluorescently and isotopically tagged PDCs across a wide range of childhood solid cancer cell lines including, Ewing sarcoma, rhabdomyosarcoma, pediatric brain tumors such as high-grade gliomas, medulloblastoma and atypical teratoid rhabdoid tumor. In subsequent testing in mouse xenograft models of neuroblastoma, Ewing sarcoma, rhabdomyosarcoma and osteosarcoma, CLR 131 provided significant benefits on tumor growth rates and survival.

"We are particularly pleased to advance CLR 131 in this refractory pediatric patient population as currently most of these children have a very poor prognosis for survival. We are highly encouraged by the preclinical data in pediatric cancers that have shown CLR 131 to have meaningful benefit on tumor growth rates and survival," stated Jim Caruso, president and chief executive officer of Cellectar Biosciences.

About CLR 131

CLR 131 is an investigational compound under development for a range of orphan designated cancers. It is currently being evaluated as a single-dose treatment in a Phase I clinical trial in patients with relapsed/refractory (R/R) multiple myeloma (MM) as well as in a Phase II clinical trial for R/R MM and select R/R lymphomas with either a one- or two-dose treatment. Based upon preclinical and interim Phase I study data, treatment with CLR 131 provides a novel approach to treating solid and hematological tumors and may provide patients with therapeutic benefits, including overall survival, an improvement in progression-free survival, surrogate efficacy marker response rate, and overall quality of life. CLR 131 utilizes the company’s patented phospholipid ether drug conjugate (PDC) tumor targeting delivery platform to deliver a cytotoxic radioisotope, iodine-131, directly to tumor cells. The FDA has granted Cellectar an orphan drug designation for CLR 131 in the treatment of MM.

About Phospholipid Drug Conjugates (PDCs)

Cellectar’s product candidates are built upon its patented cancer cell-targeting delivery and retention platform of optimized phospholipid ether-drug conjugates (PDCs). The company deliberately designed its phospholipid ether (PLE) carrier platform to be coupled with a variety of payloads to facilitate both therapeutic and diagnostic applications. The basis for selective tumor targeting of our PDC compounds lies in the differences between the plasma membranes of cancer cells compared to those of normal cells. Cancer cell membranes are highly enriched in lipid rafts, which are glycolipoprotein microdomains of the plasma membrane of cells that contain high concentrations of cholesterol and sphingolipids, and serve to organize cell surface and intracellular signaling molecules. PDCs have been tested in more than 80 different xenograft models of cancer.