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Orphan Drug Designation Request to the US Food and Drug Administration for Treatment of Ovarian Cancer

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.

3RD QUARTER RESULTS

GSK delivers Q3 sales of £7.8 billion, +4% AER, +2% CER (Press release, GlaxoSmithKline, OCT 25, 2017, View Source [SID1234521161]).

Total EPS 24.8p, +49% AER, +46% CER; Adjusted EPS 32.5p, +3% AER, flat CER

Financial highlights

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Sales growth in Pharmaceuticals and Consumer Healthcare; Vaccines sales flat

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Pharmaceuticals sales £4.2 billion +3% AER, +2% CER; Vaccines £1.7 billion +5% AER, flat at CER; Consumer Healthcare £2.0 billion +5% AER, +2% CER

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Improved Total operating margin of 23.9% (+4.9 points, including 0.2 points currency benefit) and EPS (24.8p), primarily reflecting reduced transaction-related charges related to valuations of Consumer Healthcare and HIV businesses

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Improved Adjusted Group operating margin of 31.5% (+1.0 point, no currency effect) primarily reflecting leverage from sales growth, focus on costs and benefits of restructuring. Pharmaceuticals 34.0% (-0.3 points, no currency effect); Vaccines 41.3% (+1.6 points, including 0.3 points adverse currency effect); Consumer Healthcare 20.0% (+3.9 points, including 1.3 points currency benefit)

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YTD free cash flow £1.6 billion (9 months 2016: £1.3 billion)

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19p dividend declared for quarter. Continue to expect 80p for FY 2017

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Guidance for 2017 Adjusted earnings per share growth maintained at 3% to 5% CER

Product and pipeline highlights

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New product sales of £1.7 billion, +44% AER, +40% CER, driven by continued strong performance from Tivicay/Triumeq in HIV, Relvar/Breo Ellipta and Nucala in Respiratory and meningitis vaccines

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Trelegy Ellipta approved in the US for COPD and positive opinion received in Europe. Positive results from landmark IMPACT study show benefits of Trelegy Ellipta in reducing COPD exacerbations compared to dual therapies

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Shingrix vaccine for shingles approved in US and Canada

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Phase III results for Nucala (mepolizumab) in COPD published in New England Journal of Medicine with regulatory filings planned for this year

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In Oncology, CHMP PRIME designation granted for 2857916 (BCMA antibody-drug conjugate) for relapsed and refractory multiple myeloma and new data to be presented at an upcoming scientific conference; option exercised from Adaptimmune to develop T-cell therapy (NY-ESO-1) for multiple tumour types

YmAbs Raises $50 Million in Equity Financing

On October 25, 2017 Y-mAbs Therapeutics, Inc. (YmAbs), an immunotherapy company discovering and developing innovative treatments for patients with cancer, reported that it has closed a private placement among HBM Healthcare Investments and its current shareholders raising $50 million (Press release, Y-mAbs Therapeutics, OCT 25, 2017, View Source [SID1234521160]).

YmAbs’ Founder, President and Head of Business Development and Strategy, Thomas Gad said, "We are very pleased to welcome HBM as a significant shareholder in YmAbs, together with our existing shareholders, who continue to show strong support and commitment to bringing breakthrough immunotherapies to children faced with advanced cancers and clear unmet medical needs. This new financing sets YmAbs in a strong financial position to further advance its late stage oncology pipeline."

Dr. Claus Møller, Chief Executive Officer further notes, "This funding is an important financial achievement for YmAbs, and enables us to focus on bringing our lead compounds, burtomab and naxitamab, through the final steps of the regulatory pathway towards approval."

Andreas Wicki, Chief Executive Officer of HBM Healthcare Investments added: "We are delighted to add YmAbs to our portfolio of promising healthcare companies. YmAbs’ approach to immunotherapy shows great promise and will further advance innovation and product development in the pediatric oncology field. YmAbs is a young and rapidly growing company that we are excited to support and to accompany on the way to further success."

Incyte and MacroGenics Announce Global Collaboration and Licensing Agreement for Anti-PD-1 Monoclonal Antibody MGA012

On October 25, 2017 Incyte Corporation (NASDAQ:INCY) and MacroGenics, Inc. (NASDAQ:MGNX) reported that the companies have entered into an exclusive global collaboration and license agreement for MacroGenics’ MGA012, an investigational monoclonal antibody that inhibits programmed cell death protein 1 (PD-1) (Press release, Incyte, OCT 25, 2017, View Source [SID1234521150]). Incyte has obtained exclusive worldwide rights for the development and commercialization of MGA012 in all indications, while MacroGenics retains the right to develop its pipeline assets in combination with MGA012.

“Anti-PD-1 therapy is becoming a mainstay of cancer treatment across multiple tumor types, and we believe the addition of MGA012 to our clinical pipeline is important to fulfilling our long-term development strategy in immuno-oncology. This collaboration with MacroGenics will allow us to rapidly explore the potential clinical benefit of developing MGA012 as a monotherapy and also combining anti-PD-1 therapy with several of our existing portfolio assets,” said Steven Stein, M.D., Chief Medical Officer of Incyte.

“We believe Incyte is the ideal partner for MGA012, given its immuno-oncology portfolio and dedication to researching and developing innovative and transformative cancer therapies and we hope that the combined resources of both companies will be able to significantly expand and accelerate the current development efforts for this promising molecule,” said Scott Koenig, M.D., Ph.D., President and Chief Executive Officer of MacroGenics. “Furthermore, we look forward to exploring the combination of MGA012 with multiple molecules in our own portfolio, including DART molecules for redirected T-cell killing, antibodies with enhanced effector function and ADCs, potentially to provide improved patient benefit.”

Enrollment in the dose escalation portion of the Phase 1 study of MGA012 has been completed and the molecule is currently being evaluated as monotherapy across four solid tumor types in the dose expansion portion of the study. Data from the dose escalation portion of the Phase 1 study have been accepted for poster presentation at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 32nd Annual Meeting in November 2017.

Terms of the Collaboration
Upon closing, Incyte will pay MacroGenics an upfront payment of $150 million. Incyte will receive worldwide rights to develop and commercialize MGA012 in all indications.

Per the terms of the collaboration, MacroGenics will also be eligible to receive up to $420 million in potential development and regulatory milestones, and up to $330 million in potential commercial milestones. If MGA012 is approved and commercialized, MacroGenics would be eligible to receive royalties, tiered from 15 percent to 24 percent, on future sales of MGA012 by Incyte.

Under the terms of the collaboration, Incyte will lead global development of MGA012. MacroGenics retains the right to develop its pipeline assets in combination with MGA012, with Incyte commercializing MGA012 and MacroGenics commercializing its asset(s), if any such potential combinations are approved.

In addition, MacroGenics retains the right to manufacture a portion of both companies’ global clinical and commercial supply needs of MGA012. MacroGenics intends to utilize its commercial-scale GMP facility, which is expected to be fully operational in 2018.

The transaction is expected to close in the fourth quarter of 2017, subject to the early termination or expiration of any applicable waiting periods under the Hart-Scott-Rodino Act and customary closing conditions.

Atossa Genetics Announces Preliminary Results from Phase 1 Study of Oral Endoxifen

On October 25, 2017 Atossa Genetics Inc. (NASDAQ:ATOS), a clinical-stage pharmaceutical company developing novel therapeutics and delivery methods for breast cancer and other breast conditions, reported preliminary results from its Phase 1 study of its proprietary oral Endoxifen (Press release, Atossa Genetics, OCT 25, 2017, View Source [SID1234521149]). All objectives were successfully met:

– Safety: There were no clinically significant safety signals and no clinically significant adverse events in participants receiving oral Endoxifen.

– Tolerability: Oral Endoxifen was well tolerated at each dose level and for the dosing duration utilized in the study.

– Pharmacokinetics: Oral Endoxifen demonstrated blood levels that have been associated with a therapeutic effect in the adjuvant setting in women with breast cancer.
These data demonstrate the suitability of oral Endoxifen for further clinical development.

The Phase 1 Study

The Phase 1 study was a double-blind, placebo-controlled, repeat dose study of 48 healthy female subjects. Atossa assessed safety, tolerability and the pharmacokinetics of proprietary formulations of both topical and oral Endoxifen dosage forms in varying dose levels over 28 days. The study was conducted in two parts based on route of administration. Preliminary results from the topical arm of the study were announced on September 14, 2017.

Atossa’s Proprietary Endoxifen

Endoxifen is an active metabolite of tamoxifen. Tamoxifen is an FDA-approved drug to prevent new breast cancer as well as recurrent breast cancer in breast cancer patients. Tamoxifen is a “pro-drug” meaning that it must be broken down by the liver into active compounds (metabolites), of which Endoxifen is the most active. It is these active metabolites that have the therapeutic effect.

Oral Endoxifen. Although approximately one million breast cancer survivors take tamoxifen annually, up to half of them do not fully benefit from tamoxifen, meaning they are “refractory,” for a number of reasons including that they do not properly metabolize tamoxifen into its active metabolites. Low endoxifen levels in breast cancer patients taking oral tamoxifen are associated with an increased risk of recurrence or the development of new breast tumors. Thus providing oral Endoxifen directly to the patient without having to be metabolized may help to address this problem.

Topical Endoxifen. A condition called breast density (or, MBD), typically diagnosed by a mammogram, has been shown to be an independent breast cancer risk factor. To date, 30 states require that findings of MBD be directly communicated to the patient. We believe a topical form of Endoxifen could potentially reduce MBD. Although oral tamoxifen has been shown to reduce MBD, the benefit-cost ratio is not acceptable to most physicians and their patients. For example, it is estimated that less than 5% of women at an increased risk of developing breast cancer including those with MBD take oral tamoxifen to prevent breast cancer because of the risk of, or actual side-effects of, oral tamoxifen. We are planning a Phase 2 study of topical Endoxifen in Stockholm, Sweden for the treatment of MBD.

Based on the number of women with MBD and the number of patients who have survived breast cancer but are not fully benefiting from tamoxifen, Atossa estimates that the potential markets for its proprietary oral and topical formulations of Endoxifen could potentially exceed $1 billion in annual sales.

Next Steps

“Based on these positive preliminary results, we are advancing our oral Endoxifen into Phase 2 studies,” commented Dr. Steven C. Quay, CEO and President. “We expect our initial Phase 2 study will be in women who are refractory to tamoxifen and we expect to begin that study in the first quarter of 2018,” continued Dr. Quay.

Breast Cancer Statistics

The American Cancer Society (ACS) estimates that approximately 250,000 women will be diagnosed with breast cancer in the United States this year and that approximately 40,000 will die from the disease. It is the second leading cause of cancer death in American women. Although about 100 times less common than women, breast cancer also affects men. The ACS estimates that the lifetime risk of men getting breast cancer is about 1 in 1,000; 2,470 new cases of invasive breast cancer will be diagnosed; and 460 men will die from breast cancer in 2017.

Conference Call

Atossa Genetics will host a conference call to discuss preliminary results today at 10 am Eastern time.

To listen to the call by phone, interested parties within the U.S. should call 1-844-824-3830, International callers should call 1-412-317-5140 and Canadian callers should call 1-855-669-9657. All callers should ask for the Atossa Genetics conference call. The conference call will also be available through a live webcast at www.atossagenetics.com. Details for the webcast may be found on the Company’s IR events page at View Source

A replay of the call will be available approximately one hour after the end of the call through November 24, 2017. The replay can be accessed via Atossa’s website or by dialing 877-344-7529 (domestic) or 412-317-0088 (international) or Canada Toll Free at 855-669-9658. The replay conference ID number is 10113835.