On June 26, 2017 Propanc Biopharma Inc. (OTCQB: PPCB) ("Propanc Biopharma" or "the Company"), a clinical stage biopharmaceutical company focusing on development of new and proprietary treatments for cancer patients suffering from solid tumors such as pancreatic, ovarian and colorectal cancers, reported the Company received Orphan Drug Designation (ODD) from the FDA for the use of its lead product, PRP, a solution for once daily intravenous administration of a combination of two pancreatic proenzymes trypsinogen and chymotrypsinogen, for the treatment of pancreatic cancer (Press release, Propanc, JUN 26, 2017, View Source [SID1234519693]). The approved indication is one of the most lethal malignancies with a median survival of 6 months and a 5-year survival rate of less than 5%. The lethal nature of this disease stems from its propensity to rapidly disseminate to the lymphatic system and distant organs, and is a major unmet medical issue.

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"I am extremely proud of my team for this achievement, which has been a number of years in the making," said James Nathanielsz, Propanc Biopharma’s Chief Executive Officer. "We are now firmly focused on the hard work ahead of us to proceed into First-In-Human studies as soon as possible. Receiving ODD from the FDA provides us with tremendous confidence we are on the right path, and the potential benefits to fast track the development process and receive attractive benefits for up to seven years when we achieve market approval, is very exciting for an emerging biopharmaceutical company like ours. I am truly grateful to our shareholders who continue to support our company. I hope this encourages investors to see the true value of our technology."

Under the Orphan Drug Act (ODA), drugs, vaccines, and diagnostic agents qualify for orphan status if they are intended to treat a disease affecting less than 200,000 American citizens. Under the ODA, orphan drug sponsors qualify for seven-year FDA-administered market Orphan Drug Exclusivity (ODE), tax credits of up to 50% of R&D costs, R&D grants, waived FDA fees, protocol assistance and may get clinical trial tax incentives.

"PRP is truly a unique and exciting technology, and I am really pleased we achieved this important milestone," said Dr Julian Kenyon, Propanc Biopharma’s Chief Scientific Officer. "My experience tells me that our drug has the potential to extend life meaningfully, free from the severe side effects inflicted by standard treatment approaches. Our drug is unique because it reprograms cancer cells to become benign and essentially forces them to behave as a normal cell. I look forward to seeing how PRP works in the clinic, in a controlled clinical trial."

Recent development progress for PRP includes successful completion of a GLP-compliant, 28-day repeat-dose toxicity study with no toxicological findings after administration, indicating a broad safety margin and providing sufficient data to support a safe starting dose for First-In-Human studies. The Company has also commenced development of the GMP-compliant investigational medicinal product (IMP) manufacture of PRP to support preparation of a planned clinical trial application in the UK.

Responsible for 331,000 deaths worldwide in 2012, the aggressive biology and resistance to conventional therapeutic agents leads to a typical clinical presentation of incurable disease at the time of diagnosis. Propanc Biopharma intends to introduce a new therapy which targets and eradicates cancer stem cells, the cells responsible for the aggressive dissemination, resulting in a meaningful life extension for patients.

Currently progressing towards First-In-Human studies, PRP aims to prevent tumor recurrence and metastasis from solid tumors. Eighty percent of all cancers are solid tumors and metastasis is the main cause of patient death from cancer. According to the World Health Organization, 8.2 million people died from cancer in 2012. Consequently, a report by IMS Health states innovative therapies are driving the global oncology market to meet demand, which is expected to reach $150 Billion by 2020. The Company’s initial target patient populations are pancreatic, ovarian and colorectal cancers, representing a combined market segment of $14 Billion predicted in 2020, by GBI Research.

To view Propanc Biopharma’s "Mechanism of Action" video on anti-cancer product candidate, PRP, please click on the following link: View Source

To be added to Propanc Biopharma’s email distribution list, please click on the following link: View Source and submit the online request form.

on June 26, 2017 Xenetic Biosciences, Inc. (NASDAQ: XBIO) ("Xenetic" or the "Company"), a clinical-stage biopharmaceutical company focused on the discovery, research and development of next-generation biologic drugs and novel orphan oncology therapeutics, reported that patient enrollment has commenced in its Phase 2 clinical study of XBIO-101 (sodium cridanimod) in conjunction with progestin therapy for the treatment of endometrial cancer, in a population of patients who have either failed progestin monotherapy or who have been identified as having progesterone receptor negative ("PrR-") tumors (Press release, Xenetic Biosciences, JUN 26, 2017, View Source [SID1234519692]).

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XBIO-101, Xenetic’s lead product candidate in development, is a small-molecule immunomodulator and interferon inducer which, in preliminary studies, has been shown to increase progesterone receptor ("PrR") expression in endometrial tumor tissue.

"We are very pleased to commence patient enrollment in this Phase 2 study with our flagship product candidate and believe this trial is designed to provide greater understanding of XBIO-101 for the treatment of progestin resistant endometrial cancer," stated M. Scott Maguire, Xenetic’s CEO. "Based on these preliminary findings, we believe that XBIO-101 has the potential to increase the expression of PrR on endometrial tumors thereby making them more responsive to treatment with traditional progestin therapy, and ultimately provide a much-needed solution for late stage endometrial cancer patients."

The primary objective of the open-label, multi-center, single-arm, two-period Phase 2 study is to assess the antitumor activity of XBIO-101 in conjunction with progestin therapy as measured by Overall Disease Control Rate ("ODCR") in women with recurrent or persistent endometrial carcinoma not amenable to surgical treatment or radiotherapy who have either failed progestin monotherapy or who have been identified as PrR-. Secondary objectives include assessments of efficacy and safety/tolerability parameters.

The study is expected to enroll a total of 72 women with recurrent or persistent endometrial cancer not amenable to surgical treatment or radiotherapy but suitable to be treated with progestins. All subjects determined to be PrR- at screening, as well as those subjects who experience disease progression after at least 4 weeks of progestin monotherapy, will receive XBIO-101 in combination with continued progestin treatment. Subjects will receive treatment until disease progression as defined according to RECIST 1.1 criteria.

For more information about the Phase 2 clinical study of XBIO-101 in conjunction with progestin therapy for the treatment of endometrial cancer, please visit clinicaltrials.gov and reference Identifier NCT03077698.

About Endometrial Cancer

Endometrial cancer is the most common malignancy of the female genital tract and represents a major health concern, as overall five-year survival rates have not improved over the past three decades. Annually in the United States, an estimated 60,050 patients are diagnosed with endometrial cancer and 10,470 deaths occur from this disease, representing 1.8% of all cancer deaths in the US. The incidence of endometrial cancer is on the rise with a lifetime risk of approximately 3% while the disease-specific mortality of endometrial carcinoma has been rising in the last 25 years. Endometrial cancer patients whose tumors no longer express progesterone receptors are not candidates for progestin-based therapy. Patients who fail monotherapy with progestins have no additional treatment options. XBIO-101 may improve sensitivity to progestin therapy in subjects with advanced or recurrent PrR tumors.

About XBIO-101

XBIO-101 is a small-molecule immunomodulator and interferon inducer which, in preliminary studies, has been shown to increase progesterone receptor ("PrR") expression in endometrial tissue. Restoration of PrR expression may re-sensitize endometrial tumor tissue to progestin therapy in previously unresponsive tumors.

Xenetic has commenced patient enrollment in the Phase 2 clinical study of XBIO-101 in conjunction with progestin therapy for the treatment of progestin resistant endometrial cancer, and has filed a protocol under its existing Investigational New Drug application ("IND") to expand the development of XBIO-101 into a biomarker study for the treatment of triple negative breast cancer ("TNBC").

On June 26, 2017 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), reported demonstrating responses of oral rigosertib with azacitidine in Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS), as well as oral rigosertib as a single agent (Press release, Onconova, JUN 26, 2017, View Source [SID1234519691]). The findings were presented by Onconova, Mount Sinai, and SymBio at the 22nd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) taking place on June 22-24 in Madrid, Spain.

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"Advancing oral rigosertib in the clinic as a stand-alone agent, and providing further evidence for activity of rigosertib in combination with azacitidine in patients with MDS and AML represents an extension of our pipeline," said Dr. Ramesh Kumar, CEO of Onconova. "We are positioned for multiple key milestones in 2017 and beyond, beginning with the interim analysis of our pivotal Phase 3 INSPIRE trial later this year."

Full copies of the posters and oral presentations can be accessed by visiting "Scientific Presentations" in the Investors section of Onconova’s website.

Oral Presentation: Oral Rigosertib Combined with Azacitidine in Patients with AML and MDS; Effects in Treatment Naive and Relapsed/Refractory Patients

A novel combination therapy of oral rigosertib plus injectable azacitidine was tested in this trial (09-08) at three sites in the U.S. and Europe, representing a first-in-man study of this approach. Eight AML patients were evaluable for response, with an overall response rate (ORR) of 37.5%, and responses in both secondary and refractory AML. Two additional patients had stable disease (25%). Responses were durable, with the longest response in AML approaching one year.

Among 33 evaluable MDS patients, ORR was 76%. Complete remission (CR) in eight (24%), concurrent marrow CR (mCR) and hematologic improvement (HI) in 10 (30%), mCR alone in six (18%), and HI alone in 1 (3%). ORR was 85% in hypomethylating agent (HMA) naïve patients and 62% in HMA resistant patients.

Earlier, Phase 1 and Phase 2 data in first and second-line higher risk (HR)-MDS patients were presented at the 2016 American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting and updated at the 2017 ASH (Free ASH Whitepaper) and MDS Foundation meetings. Based on these results, the authors determined that continued study in AML is warranted.

A Phase 3 study of the combination of oral rigosertib and azacitidine in patients with treatment naïve HR-MDS is currently being designed based on an end-of-phase 2 meeting with the Food and Drug Administration.

E-Poster: Rigosertib Combined with Azacitidine Epigenetically Modulates Chromatin and Hematopoietic Stem Cell Populations in MDS

Onconova’s collaborators from the Mount Sinai School of Medicine investigated the in vitro effects of rigosertib combined with azacitidine or vorinostat on two cell lines and on bone marrow samples from patients treated in the Phase 1-2 study, obtained prior to and after one cycle of the combination regimen. Azacitidine is an HMA and vorinostat is an inhibitor of Histone Deacetylase. Rigosertib’s mechanism of action is reported to be mediated by binding to a Ras Binding Domain present in Ras and its effector proteins, including PI3 Kinase and Raf. Chromatin remodeling by changes in methylation and acetylation were noted in cell-lines treated with all three agents, as well as after treatment with the two combinations. The nature of the changes induced with the two combinations was distinct.

The authors propose that rigosertib potentially functions as a chromatin modifying agent in combination with azacitidine and may overcome HMA resistance through chromatin remodeling. Rigosertib alone, and in combination, also leads to epigenetic reprogramming of hematopoietic stem cell populations (HSPCs) that may manifest in hematological improvements in the clinical setting. A U.S. patent describing the synergistic activity of rigosertib in combination with azacitidine has been issued.

SymBio, Onconova’s Partner in Japan and Korea, Presents Phase 1 data Demonstrating Oral Rigosertib as a Single Agent

E-Poster: A Multicenter, Open-label, Phase 1 Clinical Study; Safety, Efficacy, and Pharmacokinetics of Oral Rigosertib in Japanese Patients with Recurrent/Relapsed or Refractory MDS

A multicenter, open-label, Phase 1 clinical study of oral rigosertib (primary endpoint was dose-limiting toxicity) indicated that the recommended dose for a Phase 2 clinical study is 560 mg BID in a 2-out-of-3-week administration scheme in Japanese patients with recurrent/relapsed or refractory MDS. This regimen of oral rigosertib was well tolerated.

The primary endpoint of the study was dose-limiting toxicity. The secondary endpoints were 1) safety as assessed by adverse events and laboratory results, 2) efficacy as defined by the International Working Group 2006 Criteria, and 3) pharmacokinetics. Both hematological remission rate and hematological improvement rate were 11.1% of the nine patients with a median age of 70. In this study, the recommended dose was 560 mg BID. This study and a companion Phase 1 study with IV rigosertib were designed to obtain pharmacokinetics, safety, tolerability and efficacy data in MDS patients in Japan. Currently, SymBio is enrolling patients in a pivotal Phase 3 INSPIRE global study to assess the safety and efficacy of IV rigosertib.

Publication: Safety, Efficacy and Pharmacokinetics of Intravenous Rigosertib in Japanese Patients with Recurrent/Relapsed or Refractory MDS; A Multicenter, Open-label, Phase 1 Study

A multicenter, open-label, Phase 1 study of intravenous rigosertib was conducted to evaluate its safety, efficacy, and pharmacokinetics and to determine the recommended dose (RD) for Japanese patients.

The Phase 1 study showed that intravenous rigosertib (1,800 mg daily) for consecutive 72 hours was well-tolerated, indicating that this is the RD for Japanese patients with MDS, similar to a Phase 3 study in the U.S. Based on these clinical outcomes, Japanese patients with MDS are participating in a global randomized Phase 3 study to compare rigosertib with physicians’ choice of treatment.

On June 26, 2017 TG Therapeutics, Inc. (NASDAQ:TGTX), reported clinical data from two triple combination therapy trials using TGR-1202 (umbralisib), the Company’s oral, next generation PI3K delta inhibitor and TG-1101 (ublituximab), the Company’s novel glycoengineered anti-CD20 monoclonal antibody, as the backbone of the combinations (Press release, TG Therapeutics, JUN 26, 2017, View Source [SID1234519689]). The data presentations include a recap of the data from the combination of TGR-1202, TG-1101, and ibrutinib, as well as from the triple combination of TGR-1202, TG-1101, and bendamustine. Data from these trials were presented this past weekend at the 22nd European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in Madrid, Spain. These data sets were presented earlier this month at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting and/or at the 14th International Conference on Malignant Lymphoma (ICML).

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Highlights from the presentations include the following:

Oral Presentation: Chemo-free triplet combination of TGR-1202, ublituximab, and ibrutinib is well tolerated and highly active in patients with advanced CLL and NHL

This oral presentation includes data from patients with Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL) and Non-Hodgkin’s Lymphoma (NHL) treated with the triple combination of TGR-1202, TG-1101, and ibrutinib. All patients were relapsed or refractory to prior therapy, except 3 CLL patients who were treatment naïve. Three cohorts each for CLL/SLL and NHL were evaluated with TGR-1202 dose escalation starting with doses of 400 mg (cohort 1), followed by 600 mg (cohort 2) and 800 mg (cohort 3), in combination with TG-1101 at 900 mg and ibrutinib daily at 420 mg (CLL) and 560 mg (NHL).

Safety & Tolerability
Thirty-eight (38) patients were evaluable for safety (20 CLL/SLL patients, and 18 NHL patients). The triple combination appeared to be well tolerated in all patients, with neutropenia (32% all grades, 18% Grade 3/4) and pneumonia (18% all grades, 11% Grade 3/4), being the only Grade 3/4 AEs in > 10% of patients. Of the 38 patients treated to date, only two AEs (sepsis and pneumonia) led to treatment discontinuation. Median time on study was 11.1 months (range 0.4 – 30+ months) with 81% of patients on study > 6 months.

Clinical Activity
Clinical activity was observed at all dose levels with 36 of 38 patients evaluable for efficacy (19 CLL/SLL patients, and 17 NHL patients), with 2 patients having discontinued prior to first efficacy assessment (1 pneumonia, and 1 investigator discretion).

CLL/SLL Efficacy highlights include:

100% (19 of 19) Overall Response Rate (ORR), including a 32% Complete Response (CR) rate observed in patients with CLL/SLL (4 of 6 CR’s pending bone marrow confirmation)

50% of the CLL patients had a 17p and/or 11q deletion

3 CLL patients had prior BTK and/or PI3Kδ inhibitor therapy, including one patient refractory to both idelalisib and ibrutinib who attained a complete response (ongoing for 1.5+ years)
NHL Efficacy highlights include:

Response Rates observed in patients with NHL:
100% (2 of 2) ORR, including one CR in patients with Marginal Zone Lymphoma (MZL)
100% (4 of 4) ORR, including 50% CR rate in patients with Mantle Cell Lymphoma (MCL)
80% (4 of 5) ORR, including 20% CR rate in patients with Follicular Lymphoma (FL)
17% (1 of 6) ORR in patients with Diffuse Large B-cell Lymphoma (DLBCL)

FL patients were heavily pretreated including 2 with prior Autologous Stem Cell Transplant (ASCT), 1 refractory to prior ibrutinib, and 1 with 5 prior lines of rituximab based therapy

DLBCL patients had a median of 4 prior therapies, and 4 of 6 were of non-GCB subtype
Poster Presentation: Combination of TGR-1202, Ublituximab, and Bendamustine is safe and highly active in patients with advanced DLBCL and Follicular Lymphoma

This poster presentation includes data from patients with relapsed or refractory Diffuse Large B-Cell Lymphoma (DLBCL) or Follicular Lymphoma (FL) treated with the triple combination of TGR-1202 (umbralisib), TG-1101 (ublituximab), and bendamustine. Thirty-three patients were evaluable for safety of which 24 were evaluable for efficacy (9 patients were note evaluable; 7 were too early to evaluate and 2 patients were off study prior to an efficacy assessment: 1 non-related adverse event (AE) and 1 investigator decision). The triple combination appears well tolerated with no discontinuations for a treatment related AE. No events of pneumonitis and no Grade 3/4 transaminitis were reported. Twenty-one patients (64%) were refractory to prior treatment. Mean time on study was approximately 6 months.

Efficacy highlights from this poster include:

100% (4 of 4) ORR, including a 50% CR rate, observed in patients with relapsed DLBCL

50% (6 of 12) ORR, including a 42% CR rate, observed in patients with refractory DLBCL with durable CR and PR responses observed (PR on-going for > 16+ months)

88% (7 of 8) ORR, including a 50% CR rate, observed in patients with relapsed or refractory FL
PRESENTATION DETAILS:

The above referenced presentations are available on the Publications page, located within the Pipeline section, of the Company’s website at www.tgtherapeutics.com/publications.cfm.

On June 26, 2017 Atossa Genetics Inc. (NASDAQ: ATOS), a clinical-stage pharmaceutical company developing novel therapeutics and delivery methods to treat breast cancer and other breast conditions, reported that it has received a positive interim safety assessment of the first cohort receiving proprietary oral Endoxifen in its Phase 1 dose escalation study (Press release, Atossa Genetics, JUN 26, 2017, http://ir.atossagenetics.com/news/detail/816/atossa-genetics-receives-positive-safety-committee-assessment-of-first-cohort-receiving-oral-formulation-of-endoxifen-in-phase-1-dose-escalation-study [SID1234519688]). Endoxifen is an active metabolite of the FDA approved drug tamoxifen, which is indicated for breast cancer and breast cancer prevention in high-risk patients. The Independent Safety Committee reviewed the blinded data generated from the first cohort receiving the oral formulation (8 subjects) and concluded that the study may advance to the next oral dosing level.

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The study includes two arms: one receiving a topical formulation of Endoxifen and another receiving an oral formulation, each with three cohorts. The topical arm has been fully enrolled and dosed and the first of the three oral cohorts has now been dosed.

"Our Phase 1 study of our proprietary Endoxifen is progressing quickly and as planned," stated Dr. Steve Quay, CEO and President. "This interim safety determination allows us to proceed to the next dosing level and, based on the progress to date, we expect to complete enrollment in the next six weeks."

The Phase 1 study is a double-blinded, placebo-controlled, repeat dose study of 48 healthy female subjects and its objectives are to assess the pharmacokinetics of proprietary formulations of both oral and topical Endoxifen dosage forms over 28 days, as well as to assess safety and tolerability. The study is being conducted on behalf of Atossa by CPR Pharma Services Pty Ltd., Thebarton, SA, Australia.