On May 19, 2020 Clarity Pharmaceuticals, a radiopharmaceutical company focused on the treatment of serious disease, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for 64Cu-SARTATE as a diagnostic agent for the clinical management of neuroblastoma (Press release, Clarity Pharmaceuticals, MAY 19, 2020, View Source [SID1234558240]).

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"This milestone comes shortly after the FDA granted Clarity ODD for 67Cu-SARTATE, a therapy for the treatment of neuroblastoma," commented Dr Alan Taylor, Clarity’s Executive Chairman. "The grant of both diagnostic and therapeutic ODDs highlights the importance of work conducted by our Team in the field of theranostics and indicates the support for the development of SARTATE for children with cancer."

Neuroblastoma most often occurs in children younger than 5 years of age and presents when the tumour grows and causes symptoms. It is the most common type of cancer to be diagnosed in the first year of life and accounts for around 15% of paediatric cancer mortality1. High-risk neuroblastoma accounts for approximately 45% of all neuroblastoma cases. Patients with high-risk neuroblastoma have the lowest 5-year survival rates at 40%-50%.2

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. The designation allows Clarity Pharmaceuticals to qualify for a number of incentives, including: seven years of market exclusivity upon regulatory approval, if received; potential tax credits on US clinical costs; exemptions from certain administrative FDA fees; and eligibility for grants to fund future clinical work.

Dr Taylor said, "We are very excited about receiving ODDs for both the diagnostic and therapeutic applications of SARTATE in neuroblastoma and our Team is looking forward to progressing the development of both 64Cu-SARTATE and 67Cu-SARTATE in some of the leading cancer centres in the U.S. with the recruitment to our theranostic US-based Phase 1/2 trial3 expected to commence shortly."

References
Nadja C. Colon and Dai H. Chung 2011, "Neuroblastoma", Advances in Pediatrics, <View Source>
Valeria Smith and Jennifer Foster 2018, "High Risk Neuroblastoma Treatment Review", Children, <View Source>
ClinicalTrials.gov Identifier: NCT04023331

On May 18, 2020 Turning Point Therapeutics, Inc. (Nasdaq: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported the pricing of its previously announced underwritten public offering of 5,416,667 shares of its common stock at a price to the public of $60.00 per share (Press release, Turning Point Therapeutics, MAY 18, 2020, View Source [SID1234564373]). The gross proceeds to Turning Point from the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Turning Point, are expected to be approximately $325.0 million. In addition, Turning Point has granted the underwriters a 30-day option to purchase up to an additional 812,500 shares of common stock. The offering is expected to close on or about May 21, 2020, subject to satisfaction of customary closing conditions.

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Goldman Sachs & Co. LLC, SVB Leerink and Guggenheim Securities are acting as joint bookrunning managers for the offering. Wedbush PacGrow is acting as lead manager and H.C. Wainwright & Co. is acting as co-manager for the offering.

The shares of common stock described above are being offered by Turning Point pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was previously filed with and became effective by rule of the Securities and Exchange Commission (the "SEC") on May 15, 2020. A preliminary prospectus supplement and accompanying prospectus relating to the offering were filed with the SEC and are available on the SEC’s website located at View Source A final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available for free on the SEC’s website located at View Source Copies of the final prospectus supplement and the accompanying prospectus relating to the offering, when available, may be obtained from: Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, New York 10282, via telephone: 1-866-471-2526, or via email: [email protected]; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at 1-800-808-7525, ext. 6218 or by email at [email protected]; or Guggenheim Securities, LLC Attention: Equity Syndicate Department, 330 Madison Avenue, New York, NY 10017 or by telephone at 212-518-5548, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On May 18, 2020 GENFIT (Nasdaq and Euronext: GNFT), a late-stage biopharmaceutical company dedicated to improving the lives of patients with metabolic and liver diseases, reported its cash position as of March 31, 2020 and revenues for the first three months of 2020 (Press release, Genfit, MAY 18, 2020, https://ir.genfit.com/news-releases/news-release-details/genfit-reports-first-quarter-2020-financial-information-genfit [SID1234561498]).

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Cash position

As of March 31, 2020, the Company’s cash and cash equivalents amounted to €252.0 million compared with €314.1 million as of March 31, 2019 and €276.7 million as of December 31, 2019.

Revenues

Revenues for the first three months of 2020 amounted to €102 thousand compared to €1 thousand for the same period in 2019. Revenues mainly consisted of revenues from services provided to Terns Pharmaceuticals pursuant to the collaboration and license agreement in relation to their clinical trials.

Additional Information

Following the May 11, 2020 announcement of the interim results from the RESOLVE-IT Phase 3 clinical trial evaluating elafibranor in adults with NASH and fibrosis, GENFIT outlines the main operational and financial implications of this announcement:

Operational implications

Elafibranor did not show a statistically significant effect on the primary endpoint of NASH resolution without worsening of fibrosis, and therefore the top-line results do not support an application for accelerated approval by the FDA (U.S. Food and Drug Administration) under Subpart H or conditional approval by the EMA (European Medicines Agency).

However, before taking a final decision regarding the discontinuation, amendment or continuation of the RESOLVE-IT trial, GENFIT will review in detail the full dataset and will conduct additional analyses in order to:

Understand why the placebo response rate was higher than what was observed in other late stage clinical trials using similar protocols;
Determine whether there is still a potential for elafibranor in specific subpopulations.
GENFIT will then engage with the FDA and the EMA, and will take a decision regarding the discontinuation, amendment or continuation of the RESOLVE-IT trial following its discussions with regulatory authorities.

GENFIT remains fully committed to developing NIS4, its non-invasive diagnostic technology, to identify at-risk NASH patients.

With regards to PBC (Primary Biliary Cholangitis), given elafibranor’s activity in Phase 2, and its safety profile confirmed by the RESOLVE-IT interim data, and because PBC is an autoimmune disease unrelated to the metabolic origins of NASH, GENFIT is confident in its Phase 3 development program evaluating elafibranor in this indication.

Finally, GENFIT remains open to opportunities that could create value for the Company, whether through forging new strategic partnerships or new scientific collaborations.

GENFIT plans to share its updated corporate strategy in the Fall 2020, once a decision regarding the RESOLVE-IT trial is taken, including potential decisions regarding its product pipeline.

Financial implications

GENFIT is reviewing all non-essential expenses and a first series of measures includes terminating all marketing and commercialization readiness activities for elafibranor in NASH.
However, since no immediate decision can be taken regarding the future of the RESOLVE-IT trial, the trial will continue and its associated costs will continue , including those related to:
Contract Research Organization activities; and
patient monitoring, which cannot be interrupted abruptly due to ethical and regulatory concerns.
In the event a decision is taken to discontinue the RESOLVE-IT trial in the Fall 2020, given the size and complexity of the study, residual costs are to be expected and the full impact of the decision on the Company’s cash burn will not be noticeable until several months following the termination of the trial.

On May 18, 2020 Celleron reported the launch of the University of Oxford-Sichuan University Huaxi Joint Centre for Gastrointestinal Cancer, led by Prof. David Kerr (Radcliffe Department of Medicine, University of Oxford) and Prof. Li Yang (Sichuan University) (Press release, Celleron, MAY 18, 2020, View Source [SID1234560784]).

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Over the next 5 years, this new international collaboration seeks to develop an integrated cancer plan with a focus on gastrointestinal cancer, underpinned by high quality basic, translational and clinical cancer research.

The intention is to develop one team based across two sites, with an operating and governance system that will drive the exchange of ideas, which is crucial to academic development and alleviating the burden that cancer places on society across the world.

The Centre will seek to build a portfolio of multi-disciplinary teams drawing from both organizations. These teams and their projects will enable scientists and clinicians to learn from each other and apply their expertise for the benefit of cancer patients in both nations. The establishment of a bilateral researcher exchange program will drive the delivery of these.

Professor David Kerr, Co-Director of the University of Oxford-Sichuan University Huaxi Joint Centre for Gastrointestinal Cancer and CMO of Celleron Therapeutics, commented,

"The idea behind personalised cancer medicine is both simple and powerful: delivering the right treatment to the right patient at the right time. Our aim is to radically alter the way cancer is managed, saving lives by focusing on the individual"

Professor Nick La Thangue, CEO of Celleron Therapeutics, commented,

"Cancer remains a devastating and clinically unmet disease. It is very pleasing that our leadership team can contribute to international partnerships between leading universities and hospitals aiming to improve treatment of the disease".

On May 18, 2020 AXIM Biotechnologies, Inc. (OTCQB: AXIM) ("AXIM Biotech," "AXIM" or "the Company"), an international healthcare solutions company targeting oncological and cannabinoid research, reported its oncology-focused subsidiary, Sapphire Biotech, Inc. ("Sapphire"), filed a utility patent application relating to a novel biomarker for the detection of cancer in blood serum and urine (Press release, AXIM Biotechnologies, MAY 18, 2020, View Source;utm_medium=rss&utm_campaign=axim-biotechnologies-files-for-patent-for-unique-biomarker-for-early-detection-of-cancer-in-blood [SID1234558661]).

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"Sapphire’s diagnostics products have the potential to change the way in which cancer is detected and treated, not only by enabling detection with a simple blood test but also by providing an important tool for oncologists to assess the treatment protocol for patients," said AXIM Biotech CEO John W. Huemoeller II.

The patent application is based on Sapphire’s discovery of the long splice variant of the enzyme, quiescin sulfhydryl oxidase (QSOX1-L), as a biomarker in bladder cancer patients’ sera that was highly specific for the presence of cancer and was low or non-existent in normal donors’ sera. This is the first time that a clinically relevant QSOX1-L splice variant has been identified as a unique biomarker of bladder cancer and other cancers in serum.

Sapphire scientists have generated a novel antibody that selectively detects only this splice variant and have used the biomarker to develop a prototype rapid and cost-effective diagnostic test for bladder and other cancers non-invasively. On August 21, 2019, a one-year clinical trial began to evaluate Sapphire’s test to detect the QSOX1 biomarker in patients with or at risk for pancreatic cancer.

"Sapphire’s objective is to address the large mortality cancer rate by developing products using its proprietary biomarker, QSOX1, that can potentially detect cancer earlier than blood biopsy tests," said Catalina Valencia, Chief Executive Officer of Sapphire Biotech.

Sapphire’s Diagnostics Product Portfolio includes:

Universal Companion Diagnostic Test: Sapphire scientists have developed proprietary assays to detect QSOX1 levels in patients undergoing cancer treatment. The universal companion diagnostic test is intended to measure the response to treatment and to monitor post-treatment recurrence by tracking QSOX1 levels in blood. Animal studies are expected to commence imminently.
Immunohistochemistry (IHC) Diagnostic Test: The Company is developing an IHC test, using proprietary anti-QSOX1 antibodies for diagnosis of cancer. The Company’s goal is to complete the development of a commercial prototype in the first quarter of 2021.
Universal Cancer Biomarker: The Company has been studying QSOX1 levels in the blood of cancer patients to make the correlation between QSOX1 levels with various cancer stages. The ultimate goal is to validate QSOX1 as a blood biomarker for the presence of cancer in asymptomatic individuals. Breast, lung and pancreatic cancer-focused validation studies are planned.