Clarity Pharmaceuticals Announces Submission of Orphan Drug Designation Application for 64Cu-SARTATE™

On September 11, 2019 Clarity Pharmaceuticals, a radiopharmaceutical company focused on the treatment of serious disease, reported that it has submitted an Orphan Drug Designation (ODD) request with the U.S. Food and Drug Administration (FDA) for 64Cu-SARTATE, a diagnostic for the clinical management of neuroendocrine tumours (NETs) (Press release, Clarity Pharmaceuticals, SEP 11, 2019, View Source [SID1234539431]).

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NETs are a group of cancers that originates in the diffuse neuroendocrine system. They most commonly occur in the gastrointestinal tract (48%), lung (25%), and pancreas (9%), but may also occur in other areas including the breast, prostate, thymus and skin. NET patients commonly present at an advanced (metastatic) disease stage. Due to the rarity of NETs and nonspecific presentation of symptoms, a delay in diagnosis or misdiagnosis is common. Mean patient-reported time from first symptom onset to diagnosis was 52 months; 29% of patients required ≥ 5 years for a NET diagnosis, and 58% of patients had metastases at the time of diagnosis. As a result, the median survival for patients can be as short as 10 months.

FDA grants ODDs to facilitate the development of investigational therapies intended to treat, diagnose or prevent rare diseases affecting fewer than 200,000 people in the United States. An ODD provides a number of benefits to pharmaceutical development companies, including potential tax credits for clinical costs, exemptions from certain administrative FDA fees, eligibility for grants to fund future clinical work and seven years of marketing exclusivity if a marketing application is approved.

Dr Alan Taylor, Clarity’s Executive Chairman, said "At Clarity, we are continuing to progress our products to market and this is an important regulatory step for the development of 64Cu-SARTATE in NETs.

"Despite the recent advances in diagnostic methods with gallium-68 (68Ga) labelled somatostatin analogs becoming a preferred diagnostic imaging agent for well-differentiated NETs, most NETs are still diagnosed at an advanced stage and can rarely be cured in these cases.

The issues relating to 68Ga mean that many patients are not getting access to the latest diagnostic tools. 68Ga is normally eluted from expensive and inefficient generators on site which require highly trained personnel to prepare the products and maintain the generators at each treatment centre. The short half life and limited supply of 68Ga also present challenges in scheduling and scanning of patients leading to significant patient backlogs.

"64Cu-SARTATE utilises copper-64, which has a half life that allows finished product to be centrally manufactured in large volumes to meet fluctuating end-user demands without the logistical and financial challenges associated with 68Ga. 64Cu-SARTATE therefore has the potential to improve early-stage diagnosis to prolong survival. Given the superior affinity of our products to copper, 64Cu-SARTATE also has the benefit of measuring later time points compared to 68Ga-based products, which may have the added benefit of better diagnostic and therapeutic outcomes.

"The receipt of the ODD status from the US FDA would enable us to advance the development of this product more quickly and efficiently, getting Clarity closer to our ultimate goal of better treatment of children and adults with cancer."

References
Cheung, Vincent T F, and Mohid S Khan. 2015. "A Guide to Midgut Neuroendocrine Tumours (NETs) and Carcinoid Syndrome." Frontline Gastroenterology 6 (4): 264–69. View Source

Hallet, Julie, Calvin How Lim Law, Moises Cukier, Refik Saskin, Ning Liu, and Simron Singh. 2015. "Exploring the Rising Incidence of Neuroendocrine Tumors: A Population-Based Analysis of Epidemiology, Metastatic Presentation, and Outcomes." Cancer 121 (4): 589–97. View Source

2018 Raphael, Michael J., David L. Chan, Calvin Law, and Simron Singh. 2017. "Principles of Diagnosis and Management of Neuroendocrine Tumours." CMAJ : Canadian Medical Association Journal 189 (10): E398–404. View Source

ZETAGEN THERAPEUTICS, INC. AWARDED $225,000 USD GRANT FROM NIAMS FOR PHASE I STUDY OF NOVEL OSTEOINDUCTIVE LUMBAR FUSION IMPLANT FOR SMALL MOLECULE-BASED

On September 11, 2019 Zetagen Therapeutics, a private, US-based biopharmaceutical company dedicated to driving breakthrough innovation in the treatment of metastatic bone cancers and osteologic interventions, reported its award of $225,000 USD from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health (NIH) (Press release, Zetagen Therapeutics, SEP 11, 2019, View Source [SID1234643689]). The grant will be used for a Phase I validation study of a novel, surgical implant for use with the Company’s small-molecule ZetaFuse. The implant is designed to repair damaged bone through stem cell activation.

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"We are extremely pleased to be recognized by the NIAMS, allowing us to enter our next phase of research," said Joe C. Loy, CEO of Zetagen Therapeutics, Inc. "This study further builds on our earlier validation studies, allowing us to now focus on how to successfully repair damaged bone by activating the body’s own stem cells – something which can potentially be applied beyond osteolytic cancers to other orthopedic interventions."

The NIAMS grant is part of the Small Business Innovation Research Program (SBIR), a three phase award system created by the Federal Government for small businesses to engage in research and development that has the potential for commercialization and public benefit. Zetagen exclusively-licensed its platform technology from the State University of New York in 2016.

Aurora Cannabis Announces Financial Results for the Fourth Quarter and 2019 Fiscal Year

On September 11, 2019 Aurora Cannabis Inc. (the "Company" or "Aurora") (NYSE | TSX: ACB), the Canadian company defining the future of cannabis worldwide, reported its financial and operational results for the fourth quarter and fiscal year ended June 30, 2019 (Press release, Aurora Biosciences, SEP 11, 2019, View Source [SID1234539448]).

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"In 2019 Aurora took its place as the global leader in cannabis production, research, innovation, and international market development. We are executing on all our strategic priorities," said Terry Booth, CEO. "Our best in class cultivation methods allow us to grow consistent, high-quality cannabis at scale. Because of this, we’ve delivered solid revenue growth in the fourth quarter. We are working to extend our reach in the U.S. markets. Our partnership with the UFC is a basis to explore CBD-from-hemp and hemp food products. We are also exploring additional opportunities and leveraging our Strategic Advisor. We are focused on building a sustainable, high-margin business while providing patients and consumers with access to safe and reliable medicine."

Glen Ibbott, CFO, added, "We continue to see strong growth in cannabis revenues in both medical and consumer categories. Our cultivation execution continues to drive production costs lower and improve gross margins. Aurora’s diversified product portfolio remains in demand with patients and consumers alike. With the Canadian launch of derivative products in the coming months, we have made the necessary investments to ensure readiness and focus on a variety of value added products. We are very excited to supply an expanded consumer market with premium cannabis and new product forms."

Fourth Quarter 2019 Highlights

(Unless otherwise stated, comparisons are made between Fiscal Q4 2019 and Q3 2019 results and are in Canadian dollars)

Net cannabis revenue up 61% sequentially to $94.6 million
Canadian consumer cannabis revenue up 52% to $44.9 million
Medical cannabis revenue up 10% to $29.7 million
Wholesale revenues of $20.1 million
Cash cost to produce per gram sold declined 20% sequentially to $1.14 per gram in Q4 2019.

Production volume increased 86% sequentially to 29,034 kgs.

Gross margin on cannabis net revenue increased by 3% to 58% sequentially.

Aurora’s medical patient base expanded 10% to 84,729 sequentially. As at the date of this release, Aurora has approximately 89,700 active registered patients, a further increase of 6%.

Adjusted EBITDA loss of $11.7 million represents an improvement of 68% compared to $36.6 million in Q3 2019.
Subsequent Events

Closed an amended and upsized $360 million secured credit facility which includes an accordion feature that enables Aurora to upsize the facility by approximately $40 million,

Sold its remaining 28,833,334 shares of The Green Organic Dutchman Holdings Ltd ("TGOD"), at a price of $3.00 per share for aggregate gross proceeds of $86.5 million, representing an approximate 50% internal rate of return for the Company.
Full Year Fiscal 2019 Highlights

Net revenue of $247.9 million, up 349% compared to the prior year.

Gross margin on cannabis net revenue of 55% in fiscal 2019 versus 65% in fiscal 2018.

Kilograms produced and kilograms sold of 57,442 kgs and 36,628 kgs, up 920% and 629% respectively compared to fiscal 2018.
Q4 2019 Key Financial and Operational Metrics

Net revenue represents our total gross revenue cannabis products effective October 17, 2018.

These terms are defined in the "Cautionary Statement Regarding Certain Performance Measures" section of this MD&A

Refer to the following sections for reconciliation of non-GAAP measures to the IFRS equivalent measure:

Refer to the "Revenue" section for a reconciliation of cannabis net revenue to the IFRS equivalent.

Refer to the "Gross Margin" section for reconciliation to the IFRS equivalent.

Refer to the "Cash Cost of Sales of Dried Cannabis and Cash Cost to Produce Dried Cannabis Sold – Aurora Produced Cannabis"
section for reconciliation to the IFRS equivalent.

Adjusted EBITDA is calculated as net income (loss) excluding interest income (expense), accretion, income taxes, depreciation, amortization,
changes in fair value of inventory sold, changes in fair value of biological assets, share-based compensation, foreign exchange, changes in
fair value of financial instruments, gains and losses on deemed disposal, and non-cash impairment of equity investments, goodwill, and other
assets.

Represents total biological assets and cannabis inventory, exclusive of merchandise, accessories, supplies, and consumables.

During the three months ended June 30, 2019, the Company recorded non-material year-end corrections to: (i) capitalize certain payroll,
share-based compensation and borrowing costs, related to the construction of our production facilities that were incorrectly expensed in
prior periods; and (ii) reverse items that had been over-accrued in prior periods. The net impact of these adjustments to Q4 2019 Adjusted
EBITDA was a $14.9 million reduction in reported operating expenses

Consolidated net revenue increased 52% to $98.9 million in Q4 2019 as compared to $65.1 million in the prior quarter. Consumer cannabis revenues were $44.9 million in Q4 2019, an increase of 52% from the prior quarter and contributed 45% to total consolidated net revenue. Canadian medical cannabis net revenues increased to $25.2 million in Q4 2019, up 9% over the prior quarter. Revenue growth was primarily driven by additional production capacity and supply available for sale from Aurora Sky and Aurora River (Bradford).

Average net selling price of cannabis decreased by $1.08 per gram over the prior quarter from $6.40 in Q3 2019 to $5.32 in Q4 2019. This decrease is primarily attributable to the increase in sale volumes to consumer and bulk wholesale markets which yield lower average net selling prices as compared to medical markets.

Gross margin on cannabis net revenue increased to 58% in Q4 2019, compared to 55% in the prior quarter. Gross margin improvement was driven by the continued decline in cash cost to produce per gram and higher gross margins achieved on bulk sales.

During Q4 2019, Aurora produced 29,034 kilograms of cannabis as compared to 15,590 kilograms in the prior quarter. The 86.2% increase in production output was primarily due to the additional production capacity added by Aurora Sky, River (Bradford), and Ridge (Markham) facilities. Extraction capacity increased from 20,400 kilograms to 26,400 kilograms in Q4 2019. Subsequent to the quarter end, Aurora’s annual extraction capacity further increased to 45,600 kilograms.

Q4 2019 SG&A increased by 9% to $72.9 million, compared to the prior quarter. The change was primarily driven by an increase in fulfillment and shipping costs related to the growth in consumer cannabis sales and continued investment in sales initiatives, distribution network, and partnerships to conduct research, develop products, and drive brand awareness. Aurora will continue to invest in infrastructure and talent required for market share growth in the global medical and consumer cannabis markets but will remain intensely focused doing this as efficiently as possible.

In Q4 2019, adjusted EBITDA loss improved 68% to $11.7 million from $36.6 million in the prior quarter. Developing a profitable and robust global cannabis company is extremely important to Aurora. In fiscal 2019 Aurora was focused on excellence in execution, and the Company’s KPIs show its success in this regard. Furthermore, Aurora has addressed previously identified production bottlenecks and continues to see strong sell-through of the Company’s products at the retail level. However, the Canadian consumer channel continues to experience challenges at the retail level in key markets and resolution of this issue is beyond the Company’s control. Aurora is working closely with all our regulatory and channel partners to streamline distribution as the Company continues to track toward positive adjusted EBITDA on a consolidated basis.

The Company’s operating facilities current annualized run-rate production capacity is in excess of 150,000 kg per annum, based on planted rooms. As the industry leader in purpose-built cultivation, Aurora is focused on producing a consistent supply of high-quality, low-cost product to meet evolving market demand. Aurora is well-positioned to respond to market conditions quickly with shorter lead times, increased harvest cycles and high plant yields.

Outlook

The global cannabis and hemp markets represent a significant opportunity for Aurora and the Company will continue to make the necessary investments today to build long-term value for shareholders. However, Aurora will take a balanced approach to these investments with a focus on operating a sustainable and profitable business.

The introduction of new product formats to the Canadian consumer market this fall represents a significant opportunity for the Company. Aurora expects to have a robust product line-up ready to launch in December. Given the very early stage of development of the consumer market in Canada and international medical markets, management anticipates that quarter to quarter sales volumes and revenues may be volatile. The Company expects adjusted EBITDA to continue to improve in the future due to expected revenue growth, improvements in gross margin and prudent SG&A growth.

The passing of the U.S. Farm Act presents new opportunities in the largest cannabis and hemp-derived CBD market globally, and as such Aurora is committed to establishing a substantial operating footprint in the U.S. As part of the U.S. market strategy, the Company is considering its stakeholders and how various state and federal regulations will affect its business prospects. A number of alternatives to grow Aurora’s presence in the U.S. market are under evaluation and the Company is committed to only engage in activities which are permissible under both state and federal laws. Management believes there are currently market opportunities that are legal at both state and federal levels that can add operating cash flows and be critical pillars of Aurora’s strategy and long-term success.

Conference Call

Aurora will host a conference call tomorrow, September 12, 2019, to discuss these results. Terry Booth, Chief Executive Officer, Glen Ibbott, Chief Financial Officer, Cam Battley, Chief Corporate Officer, and Michael Singer, Executive Chairman, will host the call starting at 9:00 a.m. Eastern time. A question and answer session will follow management’s presentation.

Date:

Thursday, September 12th, 2019

Time:

9:00 a.m. Eastern Time | 7:00 a.m. Mountain Time

Webcast:

https://bit.ly/34gYCj5

Replay:

(416) 849-0833 or (855) 859-2056

until 12:00 midnight Eastern Time Thursday, September 19, 2019

Reference Number:

Onxeo to Attend Key Investor and Scientific Conferences

On September 11, 2019 Onxeo S.A. (Euronext Paris, NASDAQ Copenhagen: ONXEO – FR0010095596), ("Onxeo" or "the Company"), a clinical-stage biotechnology company specializing in the development of innovative drugs targeting tumor DNA Damage response (DDR) in oncology, in particular against rare or resistant cancers, reported that its management team will attend the following key investor and scientific conferences in the coming months (Press release, Onxeo, SEP 11, 2019, View Source [SID1234539466]):

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Investir Day
October 15, 2019, Paris, France

A new event providing shareholders and retail investors with the opportunity to meet and discuss with executives of listed companies. A masterclass session by Judith Greciet, CEO of Onxeo, is scheduled during the event.

The Galien Medstartup 2019
October 24, 2019, in New York, NYC, United-States

The Galien Foundation fosters, recognizes and rewards excellence in scientific innovation to improve the state of human health. Its vision is to be the catalyst for the development of the next generation of innovative treatment and technologies that will impact human health and save lives.

AACR-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper)
October 26-30, 2019, in Boston, MA, United-States

The premier international meeting featuring novel cancer therapeutics organized by the American Association for Cancer Research (AACR) (Free AACR Whitepaper), the National Cancer Institute (NCI) and the European Organisation for Research and Treatment of Cancer (EORTC).

Direct Dirigeants event with Les Echos – Investir
November 6, 2019, in Paris, France

Presentation by Judith Greciet, CEO of Onxeo, to individual investors and shareholders.

2nd Annual DNA Damage Response Therapeutics Summit
January 29-31, 2019, in Boston, MA, United-States

The Summit brings together the leading pharma and biotech companies accelerating the development of DNA repair pathway inhibitors, to optimize drug discovery, broaden patient population and generate greater efficacy.

190911_PR_Onxeo_Conferences H2 2019

ExCellThera to present at 2019 Cell & Gene Meeting on the Mesa

On September 11, 2019 ExCellThera Inc., an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, reported that Guy Sauvageau, President and CEO, will present a company update at the annual Cell & Gene Meeting on the Mesa to be held October 2-4 in Carlsbad, California (Press release, ExCellThera, SEP 11, 2019, View Source [SID1234539432]).

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ExCellThera’s lead technology, ECT-001, is a combination of a small molecule and an optimized culture system. The technology, capable of expanding stem and immune cells exponentially in as little as seven days, is used in novel curative cell therapies for patients with blood cancers and other hematologic malignancies, allowing more rapid engraftment, greatly reduced incidence of transplant-related mortality, low risk of chronic graft-versus-host disease and low risk of relapse, resulting in better outcomes for patients. ECT-001 has received FDA orphan drug designation (ODD) for the prevention of graft-versus-host disease and regenerative medicine advanced therapy (RMAT) designation in the treatment of hematologic malignancies.

The following are specific details regarding ExCellThera’s presentation at the conference:

Event: 2019 Cell & Gene Meeting on the Mesa

Date: October 2, 2019

Time: 1:45pm PT

Location: Cognate Bioservices Ballroom, Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 80 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers. Complimentary attendance at this event is available for credentialed investors and members of the media only. www.meetingonthemesa.com

A live webcast of this presentation will be available at: View Source View Source/and will also be published on the conference website shortly after the event.