On October 18, 2017 Provectus Biopharmaceuticals, Inc. (OTCQB: PVCT, www.provectusbio.com), (“Provectus” or the “Company”), a clinical-stage biotechnology company leading the development of the first small molecule oncolytic immunotherapy, intralesional (“IL”) PV-10, as a single agent for locally advanced disease as well as in combination with another agent for widely metastatic disease, both for multiple cancer indications, reported that results from the Company’s ongoing Phase 1b/2 study of IL PV-10 in combination with KEYTRUDA (pembrolizumab), Merck’s systemic anti-PD-1 (programmed death receptor-1) antibody agent, were presented at the Society for Melanoma Research 2017 Congress, held in Brisbane, Australia from October 18-21 (Press release, Provectus Pharmaceuticals, OCT 18, 2017, View Source [SID1234521017]). IL injection of PV-10 induces immunogenic cell death that results in tumor-specific reactivity in circulating CD8+ T cells.

The Phase 1b portion of the study continues to enroll patients with metastatic melanoma at clinical sites in the U.S. and Australia (NCT02557321); Stage IV patients with at least one injectable lesion who are candidates for KEYTRUDA are eligible. A total of up to 24 patients would receive the combination of IL PV-10 and KEYTRUDA every three weeks for five cycles (i.e., for up to 12 weeks, with no further PV-10 administered after week 12), followed by only KEYTRUDA every three weeks for up to 24 months. The primary endpoint for the Phase 1b trial is safety and tolerability; objective response rate and progression-free survival are key secondary endpoints (both assessed via RECIST 1.1 after five treatment cycles, and then every 12 weeks thereafter).

IL PV-10 Results from the Phase 1b Trial Presented at SMR:

Baseline characteristics (safety population, N=12): 92% men; median age of 71.5 years (range 28-81), 67% > 65 years; 67% Stage IV M1b/c.
Subject characteristics (safety population): 2.5 median number of cutaneous/subcutaneous lesions (range 1-40); patients had substantial non-injected systemic disease burden in addition to their injectable cutaneous and/or subcutaneous lesions; patients received a median of 5 cycles of PV-10 (mean 3.8, range 1-5); PV-10 was not administered after week 12.
Preliminary safety (safety population): adverse events were consistent with the established patterns for each drug; there were no unexpected toxicities or evidence of compounded toxicity.
Preliminary target lesion efficacy (efficacy evaluable population, N=10): 50% complete response; 80% objective response; maximum response.
Preliminary overall efficacy (efficacy evaluable population): 10% complete response; 50% objective response; 60% clinical benefit; highest responses observed in M1c patients; per RECIST 1.1.
Dominic Rodrigues, Chairman of the Company’s Board of Directors, said, “These preliminary results highlight the safety characteristics of the combination of intralesional PV-10 and checkpoint inhibition. The data confirm an almost complete absence of correlation of adverse events between the two drugs, which we refer to as ‘orthogonality.’ This outcome is very different from when oncolytic viruses or other biologics are used in combination with checkpoint inhibitors.”

Mr. Rodrigues added, “There also was promising clinical benefit after minimal PV-10 intervention, especially in those patients with Stage IV M1c disease. These data support the advancement of the combination of PV-10 and checkpoint inhibition in the clinical study of metastatic melanoma.”

A copy of the poster presentation is currently available on Provectus’ website at View Source

On October 18, 2017 XBiotech Inc. (NASDAQ:XBIT) reported enrollment of the first patient into a Phase I single arm study evaluating the maximum tolerated dose of OnivydeⓇ (Irinotecan liposome injection) and 5-fluorouracil/folinic acid in combination with MABp1 in a cohort of patients with advanced pancreatic adenocarcinoma and cachexia (Press release, XBiotech, OCT 18, 2017, View Source [SID1234521016]). The patient has begun treatment at Cedars-Sinai Medical Center under the care of Dr. Andrew Hendifar, the Study’s Principle Investigator, Medical Oncology lead for the Gastrointestinal Disease Research Group and Co-Director of Pancreas Oncology at Cedars-Sinai. A total of 16 patients are expected to be enrolled in the study.

Dr. Hendifar commented, “We are excited to enroll our first patient in this novel combinatorial therapy for the treatment of advanced pancreatic cancer and cachexia. This is the first attempt to add an anti-inflammatory therapy to standard chemotherapy in an effort to improve the performance, quality of life, and survival in patients with this disease.”

The study will also assess overall and progression free survival as well as evaluate the relationship between treatment tolerance and patient functional status. Various secondary measures, including changes in lean body mass, weight stability and levels of systemic inflammation will also be monitored. Onivyde will be given intravenously with MABp1 and 5-fluorouracil/folinic acid every two weeks until disease progression.

The prognosis for advanced pancreatic cancer remains poor despite decades of studies [1]. The 5-year survival has remained close to 5%, unchanged despite improvements in chemotherapeutics, surgical outcomes, and diagnostic techniques [1, 2]. Other than multi-agent cytotoxic therapy there have been no treatment advances for pancreatic cancer or its associated cachexia.

MABp1 was found to improve clusters of symptoms that included reduced pain, fatigue, improved appetite and increased lean body mass in advanced cancer patients. Patients that had these improvements were found to have less tumor progression, substantial reduced serious adverse events related to disease, and about a three-fold improvement in survival [3].

About True Human Therapeutic Antibodies
XBiotech’s True Human antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.

On October 18, 2017 Radius Health, Inc. (“Radius” or the “Company”) (Nasdaq:RDUS), reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for elacestrant, an investigational oral selective estrogen receptor down-regulator/degrader (SERD) as a treatment of women with ER+ and HER2- advanced or metastatic breast cancer (Press release, Radius, OCT 18, 2017, View Source [SID1234521010]). Fast Track designation is a process designed to facilitate the development and expedite the review of new therapies to treat serious conditions and fill unmet medical needs.

“It is estimated that about 1 in 8 women will develop invasive breast cancer over the course of their lifetime,” said Jesper Høiland, President and Chief Executive Officer of Radius Health. “If approved, elacestrant could offer a hormonal therapy alternative and potentially delay the use of chemotherapy in patients with estrogen receptor positive breast cancer. Early results of our Phase 1 trial show an encouraging efficacy and safety profile. We look forward to working closely with the FDA as we rapidly advance the development of elacestrant.”

The elacestrant clinical development program is currently ongoing with two Phase I studies in patients with ER+, HER2- advanced or metastatic breast cancer who have been heavily pre-treated (median of three prior lines of therapy) and have evaluable disease.

“The Company will provide updates on both Phase 1 studies and present two preclinical posters at the 2017 San Antonio Breast Cancer Symposium in December,” said Gary Hattersley, PhD, Chief Scientific Officer. “The FDA has indicated that, depending on the Phase 2 study results, the single-arm Phase 2 trial could be considered a pivotal study with a confirmatory study on-going at the time of NDA submission. We expect to enroll the first patient in the Phase 2 study in early 2018.”

For more information on ongoing clinical trials of elacestrant, visit www.clinicaltrials.gov.

On October 18, 2017 (GLOBE NEWSWIRE) — Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported that the U.S. Food and Drug Administration (FDA) has accepted its supplementary premarket approval (sPMA) application for BRACAnalysis CDx to be used as a companion diagnostic with AstraZeneca’s PARP inhibitor Lynparza (olaparib) in patients with HER2-negative metastatic breast cancer (Press release, Myriad Genetics, OCT 18, 2017, View Source [SID1234521008]). Myriad expects the FDA’s priority review process to conclude in the fiscal third-quarter 2018.

Myriad’s sPMA filing follows positive results from the Phase III OlympiAD trial, which demonstrated that Lynparza significantly reduced the risk of disease progression or death in patients with BRCA-mutated, HER2-negative metastatic breast cancer by 42 percent compared to standard therapy. The results of the OlympiAD trial were published in the New England Journal of Medicine in June.

“The acceptance of the sPMA for BRACAnalysis CDx is a significant step towards enabling personalized medicine for patients with metastatic breast cancer,” said Mark C. Capone, president and CEO, Myriad Genetics. “As the pioneer in companion diagnostics for PARP inhibitors, we are excited to once again partner with AstraZeneca and broaden access to Lynparza for even more patients.”

If approved, BRACAnalysis CDx would be the first and only FDA-approved companion diagnostic for use with a PARP inhibitor to identify HER2-negative metastatic breast cancer patients with a BRCA mutation who would benefit from a PARP inhibitor. The Company estimates there are approximately 125,000 patients with metastatic breast cancer who would immediately qualify for the BRACAnalysis CDx test, followed by 60,000 new patients per year on an ongoing basis.

The ongoing collaboration with AstraZeneca to develop a novel companion diagnostic test to identify candidates for treatment with olaparib began in 2007. In Dec. 2014, Myriad received FDA approval for BRACAnalysis CDx to help identify patients with advanced ovarian cancer who are eligible for fourth-line treatment with olaparib. BRACAnalysis CDx is Myriad’s first FDA-approved companion diagnostic and was the first-ever laboratory developed test approved by the FDA.

About BRACAnalysis CDx
BRACAnalysis CDx is an in vitro diagnostic device intended for the qualitative detection and classification of variants in the protein coding regions and intron/exon boundaries of the BRCA1 and BRCA2 genes using genomic DNA obtained from whole blood specimens collected in EDTA. Single nucleotide variants and small insertions and deletions (indels) are identified by polymerase chain reaction (PCR) and Sanger sequencing. Large deletions and duplications in BRCA1 and BRCA2 are detected using multiplex PCR.

Results of the test are used as an aid in identifying ovarian cancer patients with deleterious or suspected deleterious germline BRCA variants eligible for fourth line treatment with Lynparza (olaparib). This assay is for professional use only and is to be performed only at Myriad Genetic Laboratories, a single laboratory site located at 320 Wakara Way, Salt Lake City, UT 84108.

About Lynparza
Lynparza (olaparib) is an innovative, first-in-class oral poly ADP-ribose polymerase (PARP) inhibitor that may exploit tumour DNA damage response (DDR) pathway deficiencies to preferentially kill cancer cells. Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of compounds targeting DNA damage response (DDR) mechanisms in cancer cells. Lynparza is currently approved by regulatory health authorities in the EU for use as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed BRCA-mutated (germline and/or somatic) high grade serous epithelial ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy. It also is approved in the United States for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, who are in a complete or partial response to platinum-based chemotherapy and for the treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated advanced ovarian cancer who have been treated with three or more prior lines of chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for Lynparza. Lynparza is currently being investigated in another separate non-metastatic breast cancer Phase III study called OLYMPIA. This study is still open and recruiting patients internationally. In July 2017, AstraZeneca and Merck & Co., Inc., announced a global strategic oncology collaboration to jointly develop and commercialize AstraZeneca’s Lynparza, the world’s first and leading PARP inhibitor.

On October 18, 2017 Moleculin Biotech, Inc., (NASDAQ: MBRX) (“Moleculin” or the “Company”), a clinical stage pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported that 14 qualified cancer clinics have requested to participate in its clinical trial to study Annamycin for the treatment of relapsed or refractory acute myeloid leukemia (“AML”) (Press release, Moleculin, OCT 18, 2017, View Source [SID1234521006]).

“We’ve had a very positive response from the hematology oncology community,” commented Walter Klemp, Chairman and CEO of Moleculin. “With little to no cardiotoxicity and a structure that has been shown to avoid the multidrug resistance mechanisms that often defeat the current first line standard of care for AML, a number of clinicians recognize that Annamycin could provide a second chance to patients who have run out of options.”
Mr. Klemp continued: “We are pleased to have so many sites express interest in this trial, as it should help improve our chances for timely patient recruitment. In addition, seven of these interested sites are in Poland where we believe we will have access to a higher percentage of patients who are what we call ‘treatment naive’, meaning they have not been subjected to any experimental therapies before entering our trial.