On October 25, 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 it has submitted a request for Orphan Drug Designation (ODD) to the US Food and Drug Administration (FDA) for PRP, a solution for once daily intravenous administration of a combination of two pancreatic proenzymes trypsinogen and chymotrypsinogen (Press release, Propanc, OCT 25, 2017, View Source [SID1234521163]). The proposed orphan drug indication for PRP is the treatment of ovarian cancer.

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“Obtaining orphan drug designation from the FDA for our PRP therapy for ovarian cancer is a significant regulatory milestone that we are looking forward to, and will be a positive step forward in Propanc Biopharma’s ongoing efforts to develop effective treatments for metastatic cancer,” said James Nathanielsz, Propanc Biopharma’s Chief Executive Officer. “This will further reinforce our strategic investment in PRP, since we already achieved ODD status for pancreatic cancer, demonstrating significant progress in developing a potential best in class therapy that could transform treatment for patients with metastatic cancers, where limited treatment options are available. At the time the ODD is granted, we expect to be working closely with the regulatory authorities and clinical trial investigators to advance PRP promptly through the next stages of clinical development, ultimately filing a clinical trial application for First-In-Human studies in the UK early next year.”

Ovarian cancer is a disease with the lowest survival rate of all gynecological cancers, making it the seventh most common cause of cancer death in women worldwide. More than 60% of women present with stage III or stage IV metastasized cancer at the time of first diagnosis and have a five-year survival of less than 20%. The therapy is very complex and presupposes expertise in both surgery and oncology. Thus, to date therapy of ovarian cancer is a challenge and prognosis is rather poor, creating a high unmet medical need for new efficacious and safe treatment options.

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.

The rationale for developing PRP, a formulation of the pancreatic proenzymes trypsinogen and chymotrypsinogen for intravenous administration, in the proposed indication ovarian cancer is based on a set of in-vitro studies on cancer stem cells generated from various cancer cell lines as well as xenograft and orthotopic mouse models of ovarian cancer. In summary, these data indicate that the dramatic reduction of cellular markers associated with the process of epithelial-mesenchymal transition (EMT) as a consequence of PRP treatment can not only reverse the EMT process with the implication to stop tumor progression and metastasis, but also seem to repress the development of cancer stem cells (CSCs). Consequently, these results are strong indicators of the therapeutic potential of PRP that could be categorized as an anti-CSC therapeutic drug.

Further, recent scientific evidence demonstrates that EMT and CSCs play important roles in ovarian cancer chemoresistance, one of the main challenges in the treatment of recurrent ovarian cancer and responsible for the unfavourable outcome of this disease. Treatment of EMT and CSCs thus holds promise for improving current ovarian cancer therapies and prolonging the survival of recurrent ovarian cancer patients.

Preliminary early clinical data on the treatment of six patients with ovarian cancer have also been obtained using the two pancreatic proenzymes in the context of a UK “Specials” License treatment. Together, these data support the medical plausibility of the proposed indication and a distinctive benefit-safety profile of PRP for the treatment of ovarian cancer.

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 October 25, 2017 OncoSec Medical Incorporated ("OncoSec") (NASDAQ: ONCS), a company developing DNA-based intratumoral cancer immunotherapies, reported financial results for the fourth quarter and fiscal year ended July 31, 2017 (Press release, OncoSec Medical, OCT 25, 2017, View Source [SID1234521172]).

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"We have made significant progress this past quarter in advancing the development of our lead clinical program, ImmunoPulse IL-12, which we believe could provide a meaningful clinical benefit to metastatic melanoma patients with limited or no treatment options," said Punit Dhillon, President and CEO of OncoSec. "Our organization remains focused on advancing our PISCES/KEYNOTE-695 registration-directed trial to address this significant unmet medical need through an innovative accelerated pathway."

Fourth Quarter 2017 and Recent Highlights

Program Highlights and Upcoming Milestones

Presented positive Phase 2 data with ImmunoPulse IL-12 as monotherapy and in combination with pembrolizumab at the 2017 9th World Congress of Melanoma – A Joint Meeting with the Society for Melanoma Research.
50% (11/22) BORR observed at 24 weeks (42.9% [9/21] achieved RECIST v1.1 BORR).
41% (9/22) complete responders (CR), 9% (2/22) partial responders (PR), and 9% (2/22) stable disease (SD) for a total disease control rate of 59% (38.1% [8/21] achieved RECIST v1.1 durable CR) in predicted anti-PD-1 non-responder melanoma patients at 24 weeks.
Comprehensive immune monitoring data demonstrated combination of ImmunoPulse IL-12 and pembrolizumab can convert "cold" tumors to "hot" tumors, priming a coordinated innate and adaptive immune response, suggesting a synergistic relationship with anti-PD-1.
Favorable safety profile with <10% SAE as ImmunoPulse IL-12 monotherapy or in combination with pembrolizumab.
Initiated global, open-label, registration directed clinical trial, PISCES/KEYNOTE-695, of ImmunoPulse IL-12 in combination with pembrolizumab.
Enrolling patients with unresectable metastatic melanoma who have progressed or are progressing on an anti-PD-1 therapy.
Global study in the U.S. and Australia.
ImmunoPulse IL-12 granted Fast Track and Orphan Drug Designation in the U.S.
Clinical trial collaboration and supply agreement with Merck (known as MSD outside the US and Canada); attained KEYNOTE status.
Anticipate initial data mid-2018.
Late breaking poster presentation at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 32nd 2017 Annual Meeting to be held in National Harbor, MD on November 8-12, 2017.
Additional abstract highlighting preclinical data from novel multi-gene expression platform.
Presented comprehensive immune monitoring data from the Phase 2 clinical trial demonstrating that ImmunoPulse IL-12 in combination with pembrolizumab is well-tolerated and yields clinically meaningful synergy in immunologically "cold" tumors at the 2nd World Congress on Electroporation and Pulsed Electric Fields in Biology, Medicine and Food & Environmental Technologies.
Corporate Highlights

Added industry veterans Dr. Annalisa Jenkins, MBBS, FRCP and Daniel J. O’Connor to the Board of Directors;
Initiated a Technology Access Program collaboration with Jounce Therapeutics; and,
Raised and obtained commitments for $8.1 Million in offerings priced at or above market price
Fourth Quarter and Year-End 2017 Financial Results

For the fourth quarter of fiscal 2017 and the fiscal year ended July 31, 2017, OncoSec reported a net loss of $5.8 million and $21.4 million, or $0.28 per share and $1.06 per share, respectively, compared to a net loss of $6.6 million and $26.9 million, or $0.39 per share and $1.63 per share, respectively, for the same period last year. The decrease in net loss for the year ended July 31, 2017, compared with the same period in 2016, resulted primarily from: i) a $2.2 million decrease in non-cash stock-based compensation expense caused by an overall lower stock price and the Company’s tender offer exchange in December 2016 of certain then-outstanding stock options for a lesser number of new stock options with a lower exercise price; ii) a $1.8 million decrease in the costs of our research and development programs caused by our refocusing of resources to our higher priority PISCES/KEYNOTE-695 clinical program; and, iii) a $1.4 million decrease in personnel costs due to reduced headcount.

There were no revenues for the fiscal years ended July 31, 2017 or July 31, 2016.

Research and development expenses were $3.3 million and $12.0 million for the fourth quarter of fiscal 2017 and the fiscal year ended July 31, 2017, respectively, compared to $3.6 million and $14.7 million for the same periods in 2016. General and administrative expenses were $2.6 million and $9.5 million for the fourth quarter of fiscal 2017 and the fiscal year ended July 31, 2017, compared to $3.0 million and $12.1 million for the same period in 2016.

At July 31, 2017, OncoSec had $11.4 million in cash and cash equivalents, as compared to $28.7 million of cash and cash equivalents at July 31, 2016. OncoSec expects these funds to be sufficient to allow it to continue to operate its business to the third calendar quarter of 2018.

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

PISCES 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).

On October 26, 2017 Evotec AG (Frankfurt Stock Exchange: EVT, TecDAX, ISIN: DE0005664809) and TESARO, Inc. (“TESARO”) reported a three-year integrated drug discovery collaboration to discover and develop novel small molecule product candidates against an undisclosed immuno-oncology (IO) target (Press release, Evotec, OCT 25, 2017, View Source [SID1234521186]).

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Under the terms of the collaboration, Evotec will apply its integrated drug discovery platform, from lead discovery through nomination of a pre-clinical development candidate, to TESARO’s translational research pipeline to advance best-in-class oncology therapies. In particular, Evotec will leverage its industry leading structural biology platform to identify novel start points to progress into a full-blown drug discovery programme.

“TESARO is excited to work with Evotec to expand our discovery capabilities against immuno-oncology targets”, said Jeffrey Hanke, Ph.D., Executive Vice President, Research and Development, and Chief Scientific Officer of TESARO. “Evotec has a proven track record of enhancing its partners’ drug discovery efforts in oncology. We look forward to working with Evotec to accelerate the identification of new therapies to help patients facing cancer.”

Dr Mario Polywka, Chief Operating Officer of Evotec, commented: “Oncology is one of Evotec’s core therapeutic areas of focus and we are pleased to enter into this exciting and innovative partnership with TESARO, a globally recognised oncology leader and one of the fastest growing biotech companies in the USA. This collaboration further demonstrates the value of our integrated research site in Toulouse and our world-leading structural biology group in Oxford. Using our integrated drug discovery platform, we are committed to helping TESARO drive innovation in this very important field of high-unmet medical need.”
No financial details of the collaboration were disclosed.

On October 25, 2017 Actinium Pharmaceuticals, Inc. (NYSE MKT:ATNM) ("Actinium" or "the Company"), a clinical-stage biopharmaceutical company focused on developing and commercializing targeted therapies for safer myeloablation and conditioning of the bone marrow prior to a bone marrow transplant (BMT) and for the targeting and killing of cancer cells reported that the Company has successfully activated fifteen clinical trial sites in the pivotal Phase 3 SIERRA (Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia) trial (Press release, Actinium Pharmaceuticals, OCT 25, 2017, View Source [SID1234521146]).

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The SIERRA trial is planned to enroll 150 patients with relapsed or refractory acute myeloid leukemia (AML) who are age 55 and above and will compare Iomab-B and a BMT to physician’s choice of salvage chemotherapy. The primary end point is durable complete remission (dCR) of at least 6 months. Iomab-B is intended to provide safer myeloablation of the bone marrow prior to a bone marrow transplant, thus providing a potentially curative treatment option for this patient population and for patients with other leukemias, lymphomas, myelomas and other blood disorders. The following medical institutions are clinical trial sites in the Iomab-B Phase 3 clinical trials:

Center Location
MD Anderson Cancer Center Houston, Texas
Memorial Sloan Kettering Cancer Center New York, New York
Mayo Clinic Rochester, Minnesota
Mayo Clinic Jacksonville, Florida
Washington University School of Medicine Saint Louis, Missouri
Yale Cancer Center New Haven, Connecticut
Baylor Charles A. Sammons Cancer Center Dallas, Texas
The University of Kansas Cancer Center Westwood, Kansas
Roswell Park Cancer Institute Buffalo, New York
University Hospitals Cleveland Medical Center Cleveland, Ohio
The Ohio State University Comprehensive Cancer Center Columbus, Ohio
Penn State Hershey Cancer Institute Hershey, Pennsylvania
Loyola University Medical Center Maywood, Illinois
Banner MD Anderson Cancer Center Gilbert, Arizona
Fred Hutchinson Cancer Research Center Seattle, Washington
Dr. Mark Berger, Actinium’s Chief Medical Officer said, "I am delighted to be working with these world-renowned investigators and institutions in this important SIERRA trial for Iomab-B. Their interest and enthusiasm for Iomab-B further motivates my team as we work on this trial to bring Iomab-B to patients who could benefit from safer myeloablation prior to a bone marrow transplant. In many blood cancers and disorders a bone marrow transplant is the only potentially curative treatment option for patients and it is our goal to improve outcomes for these patients with Iomab-B by getting them to their transplant faster and with less complications than currently available myeloablative regimens allow. We are looking forward to adding additional clinical trial sites located in the U.S. and Canada to make this trial available to a greater range of patients and to expedite completion of the trial."

Actinium also announced that it will provide an update on the Iomab-B SIERRA trial by year end. The SIERRA trial will have three safety analyses by an independent Data Monitoring Committee when 25%, 50% and 75% patient enrollment has been reached. Also, two ad-hoc efficacy analyses may be requested by Actinium after 70 and/or 110 patients have engrafted and given enough time to achieve the primary endpoint of durable complete remission at six months post treatment.

Sandesh Seth, Actinium’s Chairman & CEO said, "Until this trial, Iomab-B had only been studied in a single center and Actinium is proud to have facilitated use of this important therapeutic option in most of the leading transplant centers in the U.S. via the SIERRA trial. These fifteen centers perform approximately a third of all AML related bone marrow transplants. Our ability to introduce Iomab-B in these centers bodes well for enrollment in the trial and also the commercial opportunity for Iomab-B as the top fifty centers account for approximately eighty percent of bone marrow transplants. There is no visible competition for Iomab-B from any drug or drug candidate that can provide safer myeloablation and enable improved outcomes of bone marrow transplant. Actinium intends to build on the clinical experience, infrastructure and supply chain capabilities that we have established thus far to complete the trial in accordance with prior guidance and, assuming a successful outcome, establish Iomab-B as the standard of care in providing safer myeloablation first in in AML and then in the other hematologic indications in which it has shown positive results."

About Iomab-B

Iomab-B is Actinium’s lead product candidate that is currently being studied in a 150-patient, multicenter pivotal Phase 3 clinical trial in patients with relapsed or refractory acute myeloid leukemia who are age 55 and above. Upon approval, Iomab-B is intended to prepare and condition patients for a bone marrow transplant, also referred to as a hematopoietic stem cell transplant, which is often considered the only potential cure for patients with certain blood-borne cancers and blood disorders. Iomab-B targets cells that express CD45, a pan-leukocytic antigen widely expressed on white blood cells with the monoclonal antibody, BC8, labeled with the radioisotope, iodine-131. By carrying iodine-131 directly to the bone marrow in a targeted manner, Actinium believes Iomab-B will avoid the side effects of radiation on most healthy tissues while effectively killing the patient’s cancer and marrow cells. In a Phase 2 clinical study in 68 patients with advanced AML or high-risk myelodysplastic syndrome (MDS) age 50 and older, Iomab-B produced complete remissions in 100% of patients and patients experienced transplant engraftment at day 28. Iomab-B was developed at the Fred Hutchinson Cancer Research Center where it has been studied in almost 300 patients in a number of blood cancer indications, including acute myeloid leukemia (AML), chronic myeloid leukemia (CML), acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), Hodgkin’s disease (HD), Non-Hodgkin lymphomas (NHL) and multiple myeloma (MM). Iomab-B has been granted Orphan Drug Designation for relapsed or refractory AML in patients 55 and above by the U.S. Food and Drug Administration and the European Medicines Agency.

On October 25, 2017 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the third quarter ended September 30, 2017 (Press release, Vertex Pharmaceuticals, OCT 25, 2017, View Source [SID1234521175]). Vertex also increased its total 2017 CF product revenue guidance, including revenue guidance for ORKAMBI (lumacaftor/ivacaftor) and KALYDECO (ivacaftor), and reiterated its total 2017 combined GAAP and non-GAAP R&D and SG&A expense guidance.

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In addition, the company today reported top-line results for three clinical studies in CF, including: a Phase 3 study of ORKAMBI in children with CF ages 2 to 5 who have two copies of the F508del mutation; a Phase 3 study of the tezacaftor/ivacaftor combination in people with CF with one copy of the F508del mutation and one copy of a gating mutation; and a Phase 2 study of the ENaC inhibitor VX-371 in combination with ORKAMBI in people with CF who have two copies of the F508del mutation.

Key financial results include:

Three Months Ended September 30,

%

2017

2016

Change

(in millions, except per share and percentage data)
ORKAMBI product revenues, net
$
336

$
234

44%
KALYDECO product revenues, net
$
213

$
176

22%
TOTAL CF product revenues, net
$
550

$
410

34%

GAAP net loss
$
(103
)

$
(39
)

n/a
GAAP net loss per share – diluted
$
(0.41
)

$
(0.16
)

n/a

Non-GAAP net income
$
136

$
43

216%
Non-GAAP net income per share – diluted
$
0.53

$
0.17

212%

"Vertex has never been stronger than it is today with significant progress across all aspects of our business," said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. "We are now treating more patients with our approved medicines than ever before, resulting in significant revenues and

earnings growth. We expect this financial trajectory to continue, driven by our pipeline of transformative CF medicines."

Dr. Leiden continued, "We look forward to continued progress in 2018 with the anticipated approval of our third CF medicine, and advancement into pivotal development of our portfolio of triple combination regimens, which have the potential to treat nearly all CF patients in the future."
Financial Highlights
Revenues:
•
Total CF net product revenues were $549.6 million compared to $409.7 million for the third quarter of 2016.
•
Net product revenues from ORKAMBI were $336.2 million compared to $234.0 million for the third quarter of 2016. The increase in ORKAMBI revenues was driven by a number of factors, including the continued uptake in children with CF ages 6 to 11 in the U.S. and the addition of revenues from European countries where ORKAMBI is currently reimbursed.
•
Net product revenues from KALYDECO were $213.5 million compared to $175.6 million for the third quarter of 2016. The increase in KALYDECO revenues was driven by the approval and uptake among people ages 2 and older in the U.S. who have certain residual function mutations.
Expenses:
•
Combined GAAP R&D and SG&A expenses were $575.7 million compared to $378.4 million for the third quarter of 2016. Combined non-GAAP R&D and SG&A expenses were $333.8 million compared to $295.0 million for the third quarter of 2016.
•
GAAP R&D expenses were $454.9 million compared to $272.4 million for the third quarter of 2016. The increase in GAAP R&D expenses was primarily due to an upfront payment of $160.0 million related to the acquisition of VX-561 (previously known as CTP-656), an investigational once-daily CFTR potentiator, from Concert Pharmaceuticals. Non-GAAP R&D expenses were $243.2 million compared to $211.0 million for the third quarter of 2016. The increase in non-GAAP R&D expenses was primarily attributable to the clinical development of the company’s triple combination regimens for CF.

•
GAAP SG&A expenses were $120.7 million compared to $106.1 million for the third quarter of 2016. Non-GAAP SG&A expenses were $90.6 million compared to $84.0 million for the third quarter of 2016. The increase in GAAP and non-GAAP SG&A expenses was driven by the global support for KALYDECO and ORKAMBI.
Net Income (Loss) Attributable to Vertex:
•
GAAP net loss was $(103.0) million, or $(0.41) per diluted share, for the third quarter of 2017, compared to a net loss of $(38.8) million, or $(0.16) per diluted share, for the third quarter of 2016. The GAAP net loss in the third quarter of 2017 was primarily due to an upfront payment of $160.0 million related to the acquisition of VX-561 from Concert Pharmaceuticals. Non-GAAP net income was $136.4 million, or $0.53 per diluted share, for the third quarter of 2017, compared to $43.1 million, or $0.17 per diluted share, for the third quarter of 2016. Third quarter 2017 non-GAAP net income growth was driven by increased CF product revenues.
Intangible Asset Impairment:
•
Based upon Phase 2 data evaluating VX-371 in combination with ORKAMBI (reported below), Vertex concluded that the intangible asset had become fully impaired, and also resulted in the deconsolidation of Parion Sciences. This impairment caused a write down of the assets, including the intangible asset, related to Parion, offset by the benefit from income taxes and the reversal of non-controlling interest, which resulted in an increase in GAAP net loss of $7.1 million for the third quarter of 2017 and had no impact on non-GAAP net income.
Cash Position:
•
As of September 30, 2017, Vertex had $1.81 billion in cash, cash equivalents and marketable securities compared to $1.43 billion in cash, cash equivalents and marketable securities as of December 31, 2016.