On November 28, 2018 Vor Biopharma, an immuno-oncology company pioneering hematopoietic stem cell (HSC) therapies for the treatment of hematological malignancies and affiliate of PureTech Health plc (LSE: PRTC), reported that the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 10,137,155 related to the Company’s technology platform (Press release, Vor BioPharma, NOV 28, 2018, View Source [SID1234531678]). This foundational patent is the first of its kind in the immuno-oncology field and broadly covers compositions and therapeutic methods related to using novel modified HSCs to enable targeted immunotherapies. The platform technology underlies Vor’s pipeline of immuno-oncology candidates, including lead candidate VOR33.

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The now patented technology is designed to address the fundamental limitations of traditional targeted therapies, including antibody-drug conjugates, bispecific antibodies, and chimeric antigen receptor (CAR) T cells, for the treatment of hematologic malignancies. These existing therapies target antigens on malignant cells that also appear on healthy cells, which can result in mass depletion of critical life-sustaining cells. Vor’s approach employs the use of antigen-modified hematopoietic stem cells (amHSCs), which are designed to repopulate a patient’s blood with cells that have a benign genetic modification to a specific surface antigen that escapes targeting and protects the cells from depletion by targeted immunotherapies. By sparing healthy blood cells, this approach may potentially improve patient safety, enable maximal dosing of cancer-targeted therapies, increase the number of potential patient populations that could benefit from these therapies, and expand the reach of targeted immunotherapies beyond B-cell cancers to a broad range of hematological malignancies, including acute myeloid leukemia (AML).

"The USPTO’s allowance of these broad claims provides validation that our approach and intellectual property are novel and first-in-class," said Aleks Radovic-Moreno, Ph.D., PureTech Health Vice President and Vor program lead. "The compositions and methods covered in the patent protect our lead product candidate as we continue development of our technology towards the clinic. This technology has the potential to provide a novel therapeutic approach for patients with aggressive blood cancers that otherwise have very few treatment options and poor prognoses."

The relevant intellectual property is exclusively licensed to Vor Biopharma and is based on technology developed by Siddhartha Mukherjee, M.D., D. Phil, Associate Professor of Medicine at Columbia University and a Staff Physician at Columbia University Medical Center, and his colleagues.

"This approach has the potential to broaden the use of targeted immunotherapies beyond B-cell cancers, and to help patients who have very limited treatment options," said Dr. Mukherjee.

About VOR33
Vor’s lead product candidate, VOR33, is designed to enable maximal CD33-targeted immunotherapy. CD33, a target that is present in the vast majority of acute myeloid leukemia (AML) patients, is also expressed in normal myeloid progenitor cells. Depletion of normal myeloid progenitor cells prevents the beneficial use of several CD33-targeted therapies at important stages throughout the treatment process, at higher doses, and for longer periods of time. By enabling new CD33-targeted therapies, VOR33 has the potential to overcome these challenges and improve treatment for AML. Vor anticipates initiating IND-enabling studies for VOR33 in early 2019.

On November 28, 2018 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported an update on its late-stage clinical development program for the Company’s proprietary galinpepimut-S (GPS) in patients with acute myeloid leukemia (AML) (Press release, Sellas Life Sciences, NOV 28, 2018, View Source [SID1234531677]).

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Following a clinical and regulatory strategy defining Type C dialogue with the U.S. Food and Drug Administration (FDA), the Company plans to proceed with a clinical study design and biostatistical plan to support a Phase 3 registrational study for maintenance therapy for AML patients who have achieved complete remission after second line (salvage) antileukemic therapy, or CR2. This study will be used as the basis for a Biologics License Application (BLA) submission, subject to results that are both statistically significant and reflective of an effect of sufficient magnitude to be clinically meaningful.

"Following discussion with the FDA, we are embarking upon a revised Phase 3 study for GPS in the monotherapy maintenance setting for AML patients who have achieved CR2. The new design is expected to streamline sample size, time to accrual completion, primary endpoint readout and potential time to market, as well as costs. We believe this new study design provides SELLAS with a quicker path to approval, provided the study is positive," said Dr. Angelos M. Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "In addition to a statistical analysis plan which we believe accords a viable pathway for meeting the primary endpoint, we have built in an adaptive design, thus further enhancing the probability of a positive study."

GPS was previously given fast track and orphan drug designations in AML by the FDA.

The planned Phase 3 registrational study will be a 1:1 randomized, open-label study comparing GPS monotherapy in the maintenance setting to investigators’ choice best available treatment (BAT) in AML patients who have achieved hematologic complete remission, with or without thrombocytopenia (CR2/CR2p), after second-line antileukemic therapy and who are deemed ineligible for or unable to undergo allogeneic stem-cell transplantation.

The study is expected to enroll approximately 116 patients at around 50 clinical sites in the United States and Europe. It is powered at 90% to show a statistically significant difference in the primary endpoint of overall survival (OS) from the time of study entry. Secondary endpoints to be measured include leukemia-free survival, antigen-specific T-cell immune response dynamics, measurable residual disease by multigene array, and assessments of AML clonal evolution and inflammasome molecular signatures in the tumor microenvironment in bone marrow biopsy samples. The study will have a planned interim analysis for safety and futility after 80 events.

This streamlined CR2 study design, as compared to the previously planned study in AML patients who achieved complete remission following first-line antileukemic therapy (CR1), substantially reduces the study size (116 patients in CR2 vs. 390 patients in CR1) and time until topline data (up to 2.5 years in CR2 vs. 4.5 years in CR1) which will result in corresponding significant cost savings. A Phase 2a study of GPS in the AML CR2 setting conducted at the Moffitt Cancer Center previously demonstrated a clinically meaningful and statistically significant three-fold OS prolongation in patients receiving GPS when compared to a comparable group of contemporaneously assessed unvaccinated patients with a median OS of 16.3 months vs 5.4 months and a p-value of 0.0175, respectively, with treatment-related adverse events primarily comprised of grade 1 or 2 local injection site reactions and only one grade 3 (transient leukopenia) adverse event. A prior Phase 2 study of GPS in AML patients who achieved CR1 also met its primary endpoint with an OS rate at 3 years from first vaccination of 47%.

"We are excited to begin this late-stage Phase 3 program with GPS in AML. Earlier studies have positioned this agent to be a potentially effective approach in prolonging survival by delaying or preventing recurrence in patients in complete remission, most of whom harbor measurable residual disease and have a poor prognosis if they are unable to undergo allotransplant. We are hopeful that this new immunotherapeutic vaccine approach will improve outcomes in this patient population, which is at a very high risk of leukemic relapse," said Hagop M. Kantarjian, MD, Professor and Chair of the Department of Leukemia at the University of Texas MD Anderson Cancer Center, and principal investigator of the upcoming Phase 3 AML clinical development program.

On November 28, 2018 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that it launched the antitumor agent trastuzumab BS for intravenous drip infusions 60 mg and 150 mg "Daiichi Sankyo" (generic name: trastuzumab (genetical recombination)[trastuzumab biosimilar 2], hereafter, "the product") in Japan today (Press release, Daiichi Sankyo, NOV 28, 2018, View Source [SID1234531676]).

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The product is a pharmaceutical agent developed by Amgen Inc. (Headquarters: Thousand Oaks, CA, U.S.A; hereafter, "Amgen") as a biosimilar product to the anti-HER2 antibody, trastuzumab. The product was approved on September 21, 2018, and indicated for breast cancer with HER2 overexpression (Dosage A), and unresectable advanced or relapsed gastric cancer with HER2 overexpression (Dosage B).

Based on the exclusive agreement on the commercialization of biosimilars concluded with Amgen in July 2016, Daiichi Sankyo is responsible for the distribution and commercialization of the product in Japan, while Amgen is responsible for its manufacture.

The product is the first biosimilar product launched by Daiichi Sankyo, and the company expects that the product will provide patients and medical professionals with various options for cancer treatment, thereby further contributing to medical treatment.

Product name

Trastuzumab BS for intravenous drip infusions 60 mg and 150 mg "Daiichi Sankyo"

Generic name

Trastuzumab (Genetical Recombination)[Trastuzumab Biosimilar 2]

Indications

Breast cancer with HER2 overexpression

Unresectable advanced or recurrent gastric cancer with HER2 overexpression

Dosage and administration

・Dosage A is applied for breast cancer with HER2 overexpression.

・Dosage B in combination with other antineoplastic agents is applied for unresectable advanced or recurrent gastric cancer with HER2 overexpression.

Dosage A: In adults 4 mg/kg (body weight) trastuzumab (recombinant) [biosimilar 2 of trastuzumab] is intravenously infused once daily at the first administration, and 2 mg/kg for 90 minutes or longer at one-week intervals for the second and subsequent treatments.

Dosage B: In adults 8 mg/kg (body weight) trastuzumab (recombinant) [generic product 2 of trastuzumab] is intravenously infused once daily at the first administration, and 6 mg/kg for 90 minutes or longer at three-week intervals for the second and subsequent treatments.

If the initial dose is well tolerated, the administration time can be shortened up to 30 minutes for the second and subsequent treatments.

Date of approval for manufacturing and marketing

September 21, 2018

Date of listing in the NHI reimbursement

November 28, 2018

Day of launch

November 28, 2018

Marketing authorization holder

DAIICHI SANKYO COMPANY, LIMITED

Partner

Amgen Inc.

On November 28, 2018 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported that data from an independent test in animal models confirmed, as previously believed, that its immuno-stimulating STAT3 inhibitor achieves disproportionately high accumulation in the pancreas (Press release, Moleculin, NOV 28, 2018, View Source [SID1234531674]).

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"Our own sponsored research suggested that WP1732 might be an ideal candidate for treating pancreatic cancer," commented Dr. Donald Picker, Moleculin’s Chief Science Officer. "Now, we have independent testing with radiolabeled drug confirming this in animal models. The propensity for such enhanced pancreatic distribution could be highly beneficial for a new pancreatic cancer drug."

Walter Klemp, Moleculin’s Chairman and CEO added, "Published research shows that the growth and survival of pancreatic cancer requires activated STAT3 (p-STAT3) and our own research suggests that WP1732 may be one of the most effective inhibitors of p-STAT3 that has demonstrated activity in in vivo models. Confirming the disproportionately high accumumulation of WP1732 in the pancreas puts us one step closer to introducing an entirely new approach to treating pancreatic cancer. This is very encouraging and confirms the direction that Moleculin has taken with WP1732. We are heavily engaged in preparing the data necessary for an Investigational New Drug (IND) application with the FDA which we are targeted to file in 2019."

On November 28, 2018 MabVax Therapeutics Holdings, Inc. (OTC Pink: MBVX), a clinical-stage biotechnology company focused on the development of antibody-based products to address unmet medical needs in the treatment of cancer and pancreatitis, reported that the Company had entered into an equity purchase agreement with Triton Funds LP, a Delaware limited partnership ("Triton Funds"), in which Triton Funds has agreed to purchase up to $1,000,000 of the Company’s to be designated Series P Convertible Preferred Stock, par value of $0.01 per share (the "Series P Preferred Stock"), subject to the effectiveness of an S-1 registration statement that the Company filed with the Securities and Exchange Commission (the "SEC") on November 20, 2018, that registers shares of common stock upon conversion of the Series P Preferred Stock (the "Equity Purchase Agreement") (Press release, MabVax, NOV 28, 2018, View Source [SID1234531673]). Under the terms of the Equity Purchase Agreement, Triton Funds is able to convert the Series P Preferred Stock into common stock at a rate based on the 5-day volume-weighted average price prior to the conversion date, or the stated market purchase price, times 75%. In addition, we agreed to issue 175,000 shares of common stock to Triton Funds LLC, manager of Triton, upon execution of the Equity Purchase Agreement to support the cost of the student-run fund. The summary above is subject to and qualified in its entirety to the Company’s filing of a registration statement on Form S-1 which can be found at:

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View Source

Dmitriy Slobodskiy Jr, Research Analyst of Triton Funds, stated, "Here at Triton Funds we strive to locate and invest into companies that make a meaningful impact on significant unmet medical needs. MabVax Therapeutics is one of the cases where a company is developing novel treatments for deadly and debilitating diseases of the pancreas, a small organ that plays a large role in human health."

Sam Yaffa, Founder of Triton Funds, stated, "One of the key factors we look for before we invest is how effective is the management team in advancing the company’s technology, and the team at MabVax has proved to be persistent in its efforts to provide benefits to patients in need of new therapies for unmet medical needs."

President and CEO J. David Hansen, stated, "We are pleased and honored that Triton Funds became interested in MabVax and its clinical stage human antibody development programs for the diagnosis and treatment of pancreatic cancer, and more recently for the development and testing of a clinical stage antibody for the treatment of pancreatitis. We intend to use these proceeds for developing a proof of concept clinical trial protocol for the treatment of pancreatitis using the Company’s antibody designated as MVT-5873 and for general corporate and working capital purposes."