On October 8, 2020 CNIO – Spanish National Cancer Research Centre reported that The CRISPR / Cas9 gene editing tool is one of the most promising approaches to advancing treatments against genetic diseases – including cancer – an area of ​​research in which progress is constantly being made (Press release, CNIO -nish National Cancer Research Centre, OCT 8, 2020, cnio.es/noticias/publicaciones/reprograman-crispr-en-ratones-para-eliminar-celulas-tumorales-sin-afectar-sanas/ [SID1234568223]). Now, the Molecular Cytogenetics Unit headed by Sandra Rodríguez-Perales at the National Center for Oncological Research (CNIO) has taken another step to be able to effectively apply this technology to eliminate so-called fusion genes , which in the future could open the doors to the development of cancer therapies that specifically eliminate tumors without affecting healthy cells. The work is published inNature Communications .

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Fusion genes are the aberrant result of the erroneous union of DNA fragments that come from two different genes, an alteration that occurs by chance during the process of cell division. If the cell cannot take advantage of the mistake, the carrier cells will die and these genes will be eliminated. But when they confer a proliferative or survival advantage, the carrier cell multiplies and the fusion genes and the proteins that they encode become the initiating event of a cancer. "Many chromosomal rearrangements and the fusion genes they produce are the origin of childhood sarcomas and leukemias," explains Sandra Rodríguez-Perales, co-lead author of the study now published by the CNIO. Also foundin tumors of the prostate, breast, lung or brain, among others: in total, up to 20% of all cancers.

Because they are only present in tumor cells, fusion genes arouse great interest in the scientific community, to turn them into highly specific therapeutic targets, in such a way that they only act on the tumor and do not produce effects in healthy cells.

And here comes the CRISPR technology. Using this technology, researchers can target specific sequences in the genome and, as if they were using molecular scissors , cut and paste DNA fragments and thus modify the genome in a controlled way. In the study carried out by the CNIO team, the researchers have worked with cell lines and mouse models of Ewing’s sarcoma and chronic myeloid leukemia , in which they have managed to eliminate tumor cells by cutting the fusion genes, the beginning of the tumor.

The tumor cell repairs itself… and destroys itself

It is the first time that CRISPR has been successfully applied for the selective removal of fusion genes in tumor cells. Previous strategies of other research teams are based on modifying the junction point of the two genes involved in the fusion to introduce a DNA sequence that induces cell death. The problem is that the introduction of external sequences has proven to be very ineffective in eliminating tumors.

CNIO researchers have used an entirely different approach to induce the tumor cell to self-destruct. "Our strategy has consisted in making two cuts in introns, non-coding regions of the gene, located at both ends of the fusion gene," explains Raúl Torres-Ruiz , co – author of the work. "In this way, the cell, when trying to repair these breaks by itself, will join the cut ends, producing the complete elimination of the fusion gene that is located in the middle." As this gene is essential for its survival, this repair automatically induces the death of the tumor cell .

"In the next steps, we will continue to carry out studies to analyze the safety and efficiency of our approach," continues Rodríguez-Perales. "These steps are essential to know if our approach could be transferred in the future to a potential clinical treatment. Furthermore, to study whether our strategy, which we have already seen to work in Ewing’s sarcoma and chronic myeloid leukemia, is also effective in other types of cancer caused by fusion genes and for which there are currently no effective therapies ", he concludes.

The work has been funded by the Ministry of Science and Innovation, the Carlos III Health Institute, the European Regional Development Fund, the Spanish Association Against Cancer (AECC), the European Research Council, the Generalitat de Catalunya, the Network of Cellular Therapy, FERO, the "la Caixa" Foundation, the Josep Carreras Foundation and the Xarxa de Bancs de Tumors de Catalunya.

On October 8, 2020 Foresee Pharmaceuticals Co., Ltd. (6576.TWO) ("Foresee") reported that the 505(b)(2) New Drug Application (NDA) for FP-001 LMIS 50mg, or CAMCEVI 42MG, a ready-to-use 6-month depot formulation of leuprolide mesylate, has been accepted for review by the U.S. Food and Drug Administration (FDA) (Press release, Foresee Pharmaceuticals, OCT 8, 2020, View Source [SID1234568241]).

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In its Day-74 letter, the FDA stated that the New Drug Application (NDA) for CAMCEVI 42MG is sufficiently complete to permit a substantive review. The goal date under the Prescription Drug User Fee Act (PDUFA) is May 27, 2021.

"This marks another important regulatory milestone for the CAMCEVI franchise," said Dr. Ben Chien, Founder and Chairman of Foresee. "We are confident that if approved, with a strong commercial partnership in the US, CAMCEVI 42MG and the CAMCEVI franchise will provide patients, as well as healthcare providers, a safe and easy-to-use treatment option with its differentiated ready-to-use profile. We look forward to the successful launch of the CAMCEVI franchise."

The NDA submission for CAMCEVI 42MG is supported by a previously communicated successful Phase 3 clinical study in 137 Advanced Prostate Carcinoma patients, where treatment with CAMCEVI 42MG injection every 6 months was demonstrated to be effective, safe and well tolerated.

On October 8, 2020 Philogen S.p.A., a clinical-stage biotechnology company focused on antibody-based therapeutics, reported that the publication of a peer-reviewed scientific article in Science Translational Medicine (Press release, Philogen, OCT 8, 2020, View Source [SID1234568206]). The article is entitled: "Immunocytokines are a promising immunotherapeutic approach against glioblastoma" (T. Weiss, E. Puca, M. Silginer, T. Hemmerle, S. Pazahr, A. Bink, M. Weller, D. Neri, P. Roth, Immunocytokines are a promising immunotherapeutic approach against glioblastoma. Sci. Transl. Med. 12, eabb2311 (2020)) and can be accessed here.

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The findings of the study show how Philogen’s proprietary antibody-cytokine fusions, called immunocytokines, demonstrated striking single-agent anti-cancer activity in immunocompetent preclinical models bearing orthotopic glioblastoma. Moreover, the treatment induced long-term tumor eradications in a proportion of the treated animals. These findings were of particular relevance as preclinical models with glioblastoma cannot be cured by any current standard of care.

The article also discusses initial clinical results of the first patients recruited in the PH-L19TNFGLIO-02/18 Phase I/II trial. Early findings showed that Philogen’s proprietary treatment, onkekafusp alpha (L19TNF), when used as a monotherapy induced not only a selective tumor necrosis in all patients (evidenced by contrast-enhanced and perfusion Magnetic Resonance Imaging), but also provided cases of prolonged disease stabilization. During the study, the treatment was shown to be safe and well tolerated.

The ongoing Phase I/II clinical study (PH-L19TNFGLIO-02/18, View Source) is investigating the use of L19TNF as a single-agent for the treatment of high-grade glioma at first recurrence/relapse, a patient population with an otherwise very poor prognosis. TNF is delivered to cancer lesions by the L19 antibody specific to the EDB domain of Fibronectin, a marker for angiogenesis that is expressed in virtually all patients with malignant glioma. The Company has previously demonstrated by nuclear medicine procedures in more than 50 patients, that the L19 antibody could efficiently localize in primary and secondary brain tumors.

The phase I/II study is open label, in subjects with glioblastoma at first recurrence/relapse and will be conducted in two parts: (i) a dose finding part to determine the recommended dose of L19TNF (the data of this part of the study are published in the paper), (ii) followed by a signal seeking part that investigates first signs of activity.

"We are very excited about the excellent preclinical results obtained in collaboration with Philogen, which is a leader in the field of targeted delivery of cytokine therapeutics. The emerging clinical results of the ongoing study with L19TNF in glioblastoma – the most malignant brain tumor – provides hope for an alternative therapeutic opportunity for patients suffering from this terrible disease. In a next step, we aim at understanding the potential of L19TNF by investigating the product in larger randomised trials." commented Prof. Michael Weller, Chairman of the Department of Neurology at the University Hospital in Zurich.

Prof. Dario Neri, Co-Chief Executive Officer of Philogen, added: "Our treatment is demonstrating effectiveness at switching the immunologically cold glioma microenvironment into a hot one, enabling effective antitumor immunity. We are delighted with the progress being made with our immunocytokine and are pleased with the great promise this could bring to patients with malignant brain tumors."

The clinical study is led by Prof. Michael Weller, Dr. Patrick Roth and Dr. Tobias Weiss at the Department of Neurology of the University of Zurich. Philogen acts as the sponsor of the trial.

On October 8, 2020 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported that it has commenced an underwritten public offering of $150,000,000 of shares of its common stock (Press release, SpringWorks Therapeutics, OCT 8, 2020, View Source [SID1234568224]). All of the shares in the offering will be sold by SpringWorks. In addition, SpringWorks expects to grant the underwriters a 30-day option to purchase up to an additional $22,500,000 of shares of its common stock in the offering. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC and Cowen and Company, LLC are acting as joint book-running managers for the offering. Wedbush Securities Inc. is acting as co-manager for the offering.

An automatic shelf registration statement on Form S-3ASR relating to these securities has been filed with the Securities and Exchange Commission (SEC) and has become effective. 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 jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

The offering will be made only by means of a prospectus and prospectus supplement that form part of the automatic shelf registration statement. A copy of the preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and may be obtained, when available, from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1115 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at [email protected]; Goldman Sachs & Co., Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526 or by emailing [email protected] or Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY,11717, Attn: Prospectus Department, by telephone at (833) 297-2926 or by emailing [email protected].

On October 8, 2020 eHealth, Inc. (NASDAQ: EHTH), a leading private online health insurance exchange, reported that the company plans to release third quarter 2020 financial results on October 22, 2020 (Press release, eHealth Insurance, OCT 8, 2020, View Source [SID1234568242]).

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Chief Executive Officer Scott Flanders and Chief Financial Officer Derek Yung will host the earnings conference call beginning at 5 p.m. Eastern Time on October 22nd to discuss these results.

Individuals interested in listening to the conference call may do so by dialing (877) 930-8066 for domestic callers and (253) 336-8042 for international callers. The participant passcode is 8351208.

A telephone replay will be available two hours following the conclusion of the call for a period of 7 days and can be accessed by dialing (855) 859-2056 for domestic callers and (404) 537-3406 for international callers. The call ID for the replay is 8351208. The live and archived webcast of the call will also be available on the company’s website at www.ehealthinsurance.com under the Investor Relations section.