On July 4, 2018 Sumitomo Dainippon Pharma Co., Ltd. (Head Office: Osaka, Japan; Representative Director, President and CEO: Hiroshi Nomura hereinafter called "Sumitomo Dainippon Pharma") reported that it submitted a new drug application for thiotepa (generic name, product code: DSP-1958) in Japan on July 3, for conditioning treatment prior to autologous hematopoietic stem cell transplantation (HSCT) for pediatric solid tumors (Press release, Sumitomo Dainippon Pharma, JUL 4, 2018, View Source [SID1234527555]).

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Thiotepa is an antitumor alkylating agent that belongs to the ethyleneimine group and inhibits DNA synthesis. In Japan, Sumitomo Chemical Co., Ltd. launched this agent as Tesupamin injection in 1958, which was then taken over by Sumitomo Pharmaceuticals Co., Ltd. (current Sumitomo Dainippon Pharma) in 1984. However, following the discontinued production of its active pharmaceutical ingredients in 2008, Sumitomo Dainippon Pharma discontinued its marketing in 2009, and the agent has not been available since then in Japan.

In Japan, thiotepa had no approved indication for conditioning treatment prior to autologous HSCT, but had been put to clinical use in combination with other chemotherapeutic agents by following the precedent set by the U.S. and Europe. In spite of the discontinuation of its marketing in 2009 in Japan, many requests for their use for this indication were made by academic societies and other parties concerned, as thiotepa was approved for conditioning treatment prior to HSCT in Europe in 2010. In response, the Evaluation Committee on Unapproved or Off-labeled Drugs with High Medical Needs of the Ministry of Health, Labour and Welfare of Japan (MHLW) determined that the
medical need for thiotepa was high. Accordingly, the MHLW invited private companies to develop the agent for the indication, to which Sumitomo Dainippon Pharma replied in September 2013 and began conducting a Phase 1 study in Japan from November 2016 as a pharmacokinetic study, the results of which are included in the data attached to this application. Meanwhile, Sumitomo Dainippon Pharma is also preparing an application for approval of thiotepa for conditioning treatment prior to autologous HSCT for malignant lymphoma.

Sumitomo Dainippon Pharma expects that thiotepa, if approved, would be a new therapeutic option for patients in areas with high unmet medical needs, such as conditioning treatment prior to autologous HSCT for pediatric solid tumors, contributing to the treatment of patients with such symptoms.

About hematopoietic stem cell transplantation (HSCT) HSCT is a powerful adjuvant therapy that aims to reconstruct hematopoietic capacity via intravenous transfusion of normal hematopoietic stem cells after eradicating intractable cancer by performing conditioning myeloablative treatment prior to transplantation using maximum levels of
anti-cancer drugs or radiation. Since patients’ own hematopoietic stem cells are collected and preserved beforehand, autologous HSCT is free from concerns about immunoreactions to transplanted hematopoietic stem cells. As such, this conditioning treatment prior to transplantation aims to eradicate tumor cells as much as possible through high-dose chemotherapy using anticancer drugs in doses that exceed the maximum tolerance of bone marrow. According to the Japanese Data Center for Hematopoietic Cell Transplantation (JDCHCT), the number of HSCT cases in Japan totaled 93,902 between 1986 and 2016, among which 33,527 cases were autologous HSCT.

About Pediatric Solid Tumors
According to the Practical guidelines for pediatric cancer 2016, approximately 2,500 new cases ofpediatric cancer occur annually in Japan, with approximately 1,300 new cases of pediatric solid tumors (excluding hematopoietic organ tumors, such as leukemia). Compared to solid tumors in adults, pediatric solid tumors demonstrate favorable chemosensitivity. As a result, better outcome of high-dose chemotherapy combined with HSCT is expected, and transplantation is now being performed as a part of everyday clinical practice. According to the JDCHCT, the number of HSCT cases for pediatric sold tumor patients totaled 3,276 between 1991 and 2016, among which 3,058
cases were autologous HSCT.

About the Evaluation Committee on Unapproved or Off-labeled Drugs with High Medical Needs
The Evaluation Committee on Unapproved or Off-label Drugs with High Medical Needs is a committee established to promote the development of unapproved or off-label drugs by pharmaceutical companies that are approved for use in Europe and the United States, etc., but not approved in Japan. It is organized by the MHLW and consists of academic experts in medical and pharmaceutical fields.

On July 3, 2018 MabVax Therapeutics Holdings, Inc. (Nasdaq: MBVX), a clinical stage oncology drug development company reported that it has granted to Y-mAbs Therapeutics, Inc., a privately held clinical stage biopharmaceutical company, an exclusive sublicense to a bi-valent ganglioside based vaccine intended to treat neuroblastoma, a rare pediatric cancer (Press release, MabVax, JUL 3, 2018, View Source [SID1234527551]). A third of neuroblastoma patients are diagnosed as infants; and ninety percent are younger than five years of age at time of diagnosis. Neuroblastoma is responsible for twelve percent of all cancer deaths in children less than 15 years of age.

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The neuroblastoma vaccine was originally developed by Dr. Philip Livingston and colleagues at Memorial Sloan Kettering Cancer Center (MSK) and licensed as part of a broader portfolio of anti-cancer vaccines to MabVax. MabVax filed for and was granted an Orphan Drug Designation for the neuroblastoma vaccine and has manufactured Phase II clinical supplies for a planned but not initiated clinical trial to be conducted with the consortium New Advances in Neuroblastoma Therapy (NANT). NANT is the only consortium of academic medical centers in the world solely dedicated to developing novel treatments and biomarkers for children with Neuroblastoma. Over the last several years MabVax has shifted its focus and resources to the Company’s human antibody discovery and development programs that are currently in early stage clinical trials and have attracted partner interest.

Y-mAbs Therapeutics, Inc ("Y-mAbs") is a clinical-stage biopharmaceutical company developing novel antibody therapeutics for oncology targets based on a range of technologies licensed from MSK under an exclusive worldwide license and research collaboration agreement. The Company has two antibody based products in advanced clinical trials for the treatment of neuroblastoma and other cancers.

Total value of the transaction to MabVax is $1.3 million plus a share of a Pediatric Disease Voucher if granted by the U.S. Food and Drug Administration ("FDA") to Y-mAbs on approval of the vaccine and the Pediatric Disease Voucher is subsequently sold. Additionally, Y-mAbs will be responsible for all further development of the product as well as any downstream payment obligations related to this specific vaccine to MSK that were specified in the original MabVax-MSK license agreement. If Y-mAbs successfully develops and receives FDA approval for the Neuroblastoma vaccine, it is obligated to file with the FDA for a Pediatric Disease Voucher. If the voucher is granted to Y-mAbs and subsequently sold, then MabVax will receive a percentage of the proceeds from the sale of the voucher by Y-mAbs.

David Hansen, President and CEO of MabVax Therapeutics, explained, "Y-mAbs is ideally positioned to continue the development of the Neuroblastoma vaccine because of their experience in immunotherapy generally and neuroblastoma specifically. If Y-mAbs is successful in development of this product, MabVax will see a greater financial benefit through participation in the sale of the Pediatric Disease Voucher."

On July 3, 2018 Eagle Pharmaceuticals, Inc. (Nasdaq:EGRX) ("Eagle" or "the Company") reported that an additional patent has been issued related to BENDEKA by the United States Patent and Trademark Office (USPTO) (Press release, Eagle Pharmaceuticals, JUL 3, 2018, View Source [SID1234527546]). Patent number 10,010,533 will expire January 2031. The USPTO has now issued or allowed a total of 15 patents in the BENDEKA family of patents expiring from 2026 to 2033.

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The newly issued patent will be listed in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) bringing Eagle’s total Orange Book listed patents for BENDEKA to thirteen1. Furthermore, as a result of a Court decision issued on June 8, 2018, BENDEKA now has Orphan Drug Exclusivity (ODE). The FDA will not be able to approve any drug applications referencing BENDEKA until the ODE expires in December 2022. Moreover, the Company now does not expect generic TREANDA entrants into the market until December 2022, rather than November 2019.

"We believe that with the recent positive ODE decision and the strength of our intellectual property portfolio, BENDEKA has longevity well beyond 2022," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.

Under a February 2015 exclusive license agreement for BENDEKA, Teva Pharmaceutical Industries, Ltd. is responsible for all U.S. commercial activities for the product including promotion and distribution.

On July 2, 2018 OSE Immunotherapeutics SA (ISIN: FR0012127173; Mnémo: OSE) received approval from the Independent Data Monitoring Committee (IDMC) to continue Atalante 1, the Company’s international pivotal Phase 3 clinical study of Tedopi for the treatment of Non-Small Cell Lung Cancer (NSCLC) following immune checkpoint inhibitor treatment (Press release, OSE Immunotherapeutics, JUL 2, 2018, View Source [SID1234528562]).

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" While checkpoint inhibitors are now considered as the standard of care in first- and second-line treatment of advanced NSCLC, there is a strong clinical need for patients in immune escape after such treatment. Our Tedopi neoepitope product is well positioned to benefit NSCLC patients experiencing treatment failure after checkpoint inhibitors, as there is currently no approved treatment for these patients," said Alexis Peyroles, CEO of OSE Immunotherapeutics.

The Tedopi Phase 3 trial, Atalante 1, is evaluating the benefit of Tedopi in HLA-A2 positive patients with NSCLC at invasive stage IIIB or metastatic stage IV, in 2nd or 3rd line treatment following failure of a checkpoint inhibitor, compared to current standard chemotherapy treatments in this patient population. The primary endpoint of the trial is overall survival. This international trial is being conducted in the U.S., in Europe and in Israel.

On July 2, 2018 DNAtrix, a leader in oncolytic virus immunotherapies for cancer, reported that Sonia Tejada, MD, PhD, neurosurgeon and investigator at Clínica Universidad de Navarra, will present updated clinical data from an ongoing trial of its oncolytic virus DNX-2401 (tasadenoturev) for pediatric diffuse midline gliomas (Press release, DNAtrix, JUL 2, 2018, View Source [SID1234527602]). The data were selected for oral presentation at the Biennial International Symposium for Pediatric Neuro-Oncology taking place June 29 – July 3, 2018 in Denver, CO.

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Diffuse intrinsic pontine glioma (DIPG), also known as diffuse midline glioma, is a rare and highly aggressive infiltrative tumor of the brainstem with the worst prognosis of any pediatric cancer. No effective treatments are available and novel treatment approaches are needed. The evaluation of oncolytic adenovirus, DNX-2401, in DIPG is based on data from clinical studies of DNX-2401 in adults with recurrent glioblastoma that demonstrate prolonged survival while maintaining a favorable safety profile compared to approved therapies.

Dr. Tejada will report that DNX-2401 can be administered safely to the pons in pediatric patients prior to radiotherapy, with minimal side effects. Safety and clinical activity will be reported to date. The presentation will also report results from preclinical studies demonstrating the synergistic antitumor activity of DNX-2401 and radiation in animal models of high grade glioma and DIPG.

"We have treated six pediatric DIPG patients with DNX-2401 and observed no grade 3 or 4 adverse events, indicating that this a safe therapy for pediatric patients with brain tumors. Based on these updated results, we intend to test DNX-2401 in a range of brain tumors that affect children," said Sonia Tejada, MD, PhD.

"DNX-2401 can be delivered safely via cannula directly into the pontine glioma, which circumvents the challenge of drugs failing to reach the target," said Frank Tufaro, PhD, CEO of DNAtrix. "Early results are encouraging."

Details of the presentation are as follows:

Oncolytic virus pHGG and DIPGs: from the bench to the bedside

Abstract Session: DIPG/Diffuse Midline Glioma

Abstract Number: DIPG-17

Presenter: Sonia Tejada, MD, PhD

Date: Monday, July 2, 2018

To access the paper describing the Phase 1 study protocol, visit the Neurosurgery website: View Source

For more information about ongoing DNAtrix clinical studies, visit the ClinicalTrials.gov website: NCT03178032 (DNX-2401 for newly diagnosed pediatric diffuse intrinsic pontine glioma) and NCT02798406 (DNX-2401 + pembrolizumab for recurrent glioblastoma).

About DNX-2401 (Tasadenoturev)
DNX-2401 is an investigational oncolytic immunotherapy designed to treat cancer. DNX-2401 sets off a chain reaction of tumor cell killing by selectively replicating within cancer cells (but not normal cells), causing tumor destruction and further spread of the oncolytic virus to adjacent tumor cells. This process then triggers an immune response directed against the tumor. DNX-2401 has been well tolerated in patients with glioblastoma and survival has been prolonged compared to other therapies.