On January 7, 2020 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, and NEC Corporation (NEC; TSE: 6701), a leader in IT and network technologies, reported that the first patients have been enrolled in the first-in-human trials evaluating TG4050, a therapeutic vaccine based on the myvac technology and powered by NEC’s cutting-edge AI capabilities (Press release, NEC, JAN 7, 2020, View Source [SID1234552727]). In these Phase 1 trials, TG4050 is being administered to patients with head and neck cancer who have a high risk of relapse after surgery and patients with ovarian cancer after surgery and adjuvant therapy.
Transgene’s highly innovative myvac technology allows the generation of a virus-based immunotherapy within a very short time frame while encoding patient-specific mutations identified and selected by NEC’s Neoantigen Prediction System.

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TG4050 has been designed to target up to 30 patient-specific neoantigens (cancer cell mutations). They are selected using NEC’s Neoantigen Prediction System, an advanced AI technology that has already been applied in the field of oncology. The prediction system is based on more than two decades of expertise in AI and has been trained on proprietary immune data, allowing it to accurately prioritize and select the most immunogenic sequences.
Transgene uses its expertise in viral vectorization via myvac to incorporate the selected neoantigen sequences in the genome of the Modified Vaccinia virus Ankara (MVA) viral vector. The Company has also set up a unique in-house good manufacturing practice (GMP) unit dedicated to the manufacturing of the individualized batches of TG4050 needed for the clinical development of this novel therapeutic vaccine.

"As each patient’s cancer is unique, we have developed a therapy that turns their solid tumor’s genetic signature into a powerful highly specific anticancer weapon. TG4050 is based on an MVA viral vector that has proven biological activity and has the ability to elicit an immune response against tumor antigens. Our partnership with NEC ensures that TG4050 is benefitting from its world-leading expertise in artificial intelligence and its unique algorithm that is used to select up to 30 patient-specific antigens that allow this novel vaccine to induce a strong immune response. We are convinced that TG4050, which is at the crossroad of immunotherapy and big data sciences, will herald the start of a new era in the fight against cancer,"
explained Philippe Archinard, Chairman and Chief Executive Officer of Transgene.

"We are excited to enroll our first patients in these trials and see TG4050 advance to the clinic. This is another step closer towards the realization of an AI-driven individualized immunotherapy for each patient. Our unique partnership with Transgene enables us to leverage its significant clinical development know-how and proven viral vector delivery platform. We are hopeful that TG4050 will make a significant difference for patients throughout the world," commented Osamu Fujikawa, Senior Vice President, NEC Corporation.

A Phase 1 clinical trial of TG4050 is enrolling patients with ovarian cancer after surgery and first-line chemotherapy. This multi-center, one-arm trial will recruit patients in the USA and in France. Endpoints of the trial include safety, feasibility and biological activity of the therapeutic vaccine. Dr. Matthew Block, MD, PhD, immunologist and medical oncologist at the Mayo Clinic, is conducting the trial in the USA; in France, the trial will be conducted by Dr. Martinez at Toulouse-Oncopole and by Pr. Le Tourneau at Institut Curie.

Another Phase 1 clinical trial of TG4050 is enrolling patients with newly diagnosed, locoregionally advanced, HPV negative, squamous cell carcinoma of the head and neck (SCCHN) that have received an adjuvant (first-line) therapy after surgery. This multi-center, open label, randomized two arms trial will include patients in the UK and in France. Patients will receive either TG4050 monotherapy after completion of the adjuvant therapy or in combination with the standard of care at the time of recurrence. Endpoints of the trial include safety, feasibility and biological activity of the therapeutic vaccine. In France, the trial is being conducted by Pr. Delord at Toulouse-Oncopole and by Pr. Le Tourneau at Institut Curie; in the UK, the trial is coordinated by Pr. Ottensmeier from Southampton University.

On January 7, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported that Michael Kauffman, MD, PhD, Chief Executive Officer, reported that it will present at the 38th Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 10:00 a.m. PT at the Westin St. Francis in San Francisco, followed by a question and answer breakout session at 10:30 a.m. PT (Press release, Karyopharm, JAN 7, 2020, View Source [SID1234552777]).

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A live webcast of the presentation and breakout session will be available on the "Events & Presentations" page in the Investors section of the Company’s website at View Source A replay of the webcasts will be archived on the Company’s website for 30 days following the presentation.

On January 7, 2020 Exelixis, Inc. (NASDAQ: EXEL) reported that based on continued encouraging efficacy and safety data, the company plans to further expand the metastatic castration-resistant prostate cancer (CRPC) cohort of COSMIC-021, the phase 1b trial of cabozantinib (CABOMETYX) in combination with atezolizumab (TECENTRIQ) in patients with locally advanced or metastatic solid tumors (Press release, Exelixis, JAN 7, 2020, View Source [SID1234552794]). The cohort, which was previously expanded from 30 to 80 patients in July 2019, will now include up to 130 patients.

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"We continue to see encouraging efficacy and safety data from the prostate cancer cohort in COSMIC-021," said Gisela Schwab, M.D., President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis. "Expanding this cohort by an additional 50 patients will allow us to further document how the combination of cabozantinib and atezolizumab may benefit this patient population as we assess our potential regulatory plans for the combination and prepare to initiate a phase 3 pivotal trial. We look forward to the presentation of initial data from this cohort of CRPC patients at ASCO (Free ASCO Whitepaper) GU 2020 in February."

Based on preliminary encouraging activity, as determined by response assessment per Response Evaluation Criteria in Solid Tumors (version 1.1) (RECISTv1.1), and safety data of patients enrolled in the metastatic CRPC cohort, 50 additional patients with metastatic CRPC (130 total) who have histologically or cytologically confirmed adenocarcinoma of the prostate are being enrolled in the trial.

Initial data from the CRPC cohort in COSMIC-021 will be presented at ASCO (Free ASCO Whitepaper) GU 2020 in San Francisco on Thursday, February 13th during Poster Session A: Prostate Cancer at 11:30 a.m. – 1:00 p.m. PT and again at 5:30 – 6:30 p.m. PT.

COSMIC-021 includes 24 cohorts and aims to enroll up to 1,732 patients with advanced or metastatic solid tumors including CRPC, renal cell carcinoma (RCC), hepatocellular carcinoma (HCC), non-small cell lung cancer (NSCLC), colorectal cancer, ovarian cancer, and urothelial carcinoma (UC), among others. The primary objective in the expansion stage of this trial is to determine the objective response rate in each cohort. More information about the currently enrolling cohorts in this trial is available at ClinicalTrials.gov. To date, early data from various cohorts of this study have informed the initiation or planned initiation of several phase 3 pivotal trials, evaluating the combination in advanced HCC, CRPC, NSCLC and RCC.

TECENTRIQ (atezolizumab) is a registered trademark of Genentech, a member of the Roche Group.

About the COSMIC-021 Study

COSMIC-021 is a multicenter, phase 1b, open-label study that is divided into two parts: a dose-escalation phase and an expansion cohort phase. The dose-escalation phase was designed to enroll patients either with advanced RCC with or without prior systemic therapy or with inoperable, locally advanced, metastatic or recurrent UC (including renal, pelvis, ureter, urinary bladder and urethra) after prior platinum-based therapy. Ultimately, all 12 patients enrolled in this stage of the trial were patients with advanced RCC. The dose-escalation phase of the study determined the optimal dose of cabozantinib to be 40 mg daily when given in combination with atezolizumab (1200 mg infusion once every 3 weeks). These results were presented at the European Society for Medical Oncology 2018 Congress.

In the expansion phase, the trial is enrolling 24 cohorts in 12 tumor types: RCC, UC, NSCLC, CRPC, HCC, triple-negative breast cancer, epithelial ovarian cancer, endometrial cancer, gastric or gastroesophageal junction adenocarcinoma, colorectal adenocarcinoma, head and neck cancer, and differentiated thyroid cancer. Up to 1,720 patients may enroll in this phase of the trial: each expansion cohort will initially enroll approximately 30 patients, and up to 10 cohorts may expand enrollment up to 1,000 additional patients in the expansion phase.

Four of the cohorts are exploratory: three are enrolling approximately 30 patients each with advanced UC, CRPC or NSCLC to be treated with cabozantinib as a single-agent, and one is enrolling approximately 10 patients with advanced CRPC to be treated with single-agent atezolizumab. Exploratory cohorts have the option to be expanded up to 80 patients (cabozantinib) and 30 patients (atezolizumab) total.

About CABOMETYX (cabozantinib)

In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC and for the treatment of patients with HCC who have been previously treated with sorafenib. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan.

Please see Important Safety Information below and full U.S. prescribing information at View Source

U.S. Important Safety Information

Hemorrhage: Severe and fatal hemorrhages occurred with CABOMETYX. The incidence of Grade 3 to 5 hemorrhagic events was 5% in CABOMETYX patients. Discontinue CABOMETYX for Grade 3 or 4 hemorrhage. Do not administer CABOMETYX to patients who have a recent history of hemorrhage, including hemoptysis, hematemesis, or melena.
Perforations and Fistulas: GastrointestinaI (GI) perforations, including fatal cases, occurred in 1% of CABOMETYX patients. Fistulas, including fatal cases, occurred in 1% of CABOMETYX patients. Monitor patients for signs and symptoms of perforations and fistulas, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a fistula that cannot be appropriately managed or a GI perforation.
Thrombotic Events: CABOMETYX increased the risk of thrombotic events. Venous thromboembolism occurred in 7% (including 4% pulmonary embolism) and arterial thromboembolism in 2% of CABOMETYX patients. Fatal thrombotic events occurred in CABOMETYX patients. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or serious arterial or venous thromboembolic event requiring medical intervention.
Hypertension and Hypertensive Crisis: CABOMETYX can cause hypertension, including hypertensive crisis. Hypertension occurred in 36% (17% Grade 3 and <1% Grade 4) of CABOMETYX patients. Do not initiate CABOMETYX in patients with uncontrolled hypertension. Monitor blood pressure regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume at a reduced dose. Discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy or for hypertensive crisis.
Diarrhea: Diarrhea occurred in 63% of CABOMETYX patients. Grade 3 diarrhea occurred in 11% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 diarrhea, Grade 3 diarrhea that cannot be managed with standard antidiarrheal treatments, or Grade 4 diarrhea.
Palmar-Plantar Erythrodysesthesia (PPE): PPE occurred in 44% of CABOMETYX patients. Grade 3 PPE occurred in 13% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 PPE or Grade 3 PPE.
Proteinuria: Proteinuria occurred in 7% of CABOMETYX patients. Monitor urine protein regularly during CABOMETYX treatment. Discontinue CABOMETYX in patients who develop nephrotic syndrome.
Osteonecrosis of the Jaw (ONJ): ONJ occurred in <1% of CABOMETYX patients. ONJ can manifest as jaw pain, osteomyelitis, osteitis, bone erosion, tooth or periodontal infection, toothache, gingival ulceration or erosion, persistent jaw pain, or slow healing of the mouth or jaw after dental surgery. Perform an oral examination prior to CABOMETYX initiation and periodically during treatment. Advise patients regarding good oral hygiene practices. Withhold CABOMETYX for at least 28 days prior to scheduled dental surgery or invasive dental procedures. Withhold CABOMETYX for development of ONJ until complete resolution.
Wound Complications: Wound complications were reported with CABOMETYX. Stop CABOMETYX at least 28 days prior to scheduled surgery. Resume CABOMETYX after surgery based on clinical judgment of adequate wound healing. Withhold CABOMETYX in patients with dehiscence or wound healing complications requiring medical intervention.
Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS, a syndrome of subcortical vasogenic edema diagnosed by characteristic finding on MRI, can occur with CABOMETYX. Evaluate for RPLS in patients presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue CABOMETYX in patients who develop RPLS.
Embryo-Fetal Toxicity: CABOMETYX can cause fetal harm. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Verify the pregnancy status of females of reproductive potential prior to initiating CABOMETYX and advise them to use effective contraception during treatment and for 4 months after the last dose.
Adverse Reactions: The most commonly reported (≥25%) adverse reactions are: diarrhea, fatigue, decreased appetite, PPE, nausea, hypertension, and vomiting.
Strong CYP3A4 Inhibitors: If coadministration with strong CYP3A4 inhibitors cannot be avoided, reduce the CABOMETYX dosage. Avoid grapefruit or grapefruit juice.
Strong CYP3A4 Inducers: If coadministration with strong CYP3A4 inducers cannot be avoided, increase the CABOMETYX dosage. Avoid St. John’s wort.
Lactation: Advise women not to breastfeed during CABOMETYX treatment and for 4 months after the final dose.
Hepatic Impairment: In patients with moderate hepatic impairment, reduce the CABOMETYX dosage. CABOMETYX is not recommended for use in patients with severe hepatic impairment.

On January 7, 2020 Cellect Biotechnology Ltd. (Nasdaq: APOP), a developer of innovative technology which enables the functional selection of stem cells, reported it received official communication from two jurisdictions, the European Patent Office (EPO) and the Israeli Intellectual Property Office, regarding their intention to grant European Patent Application No. 14851547.1 and Israeli Patent Application No. 244982, respectively (Press release, Cellect Biotechnology, JAN 7, 2020, View Source [SID1234552828]). These patent applications include a cell-based product and a method of manufacturing a stem and progenitor cell population with enhanced activity by short incubation with an apoptotic ligand.

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"Continued recognition from global jurisdictions validates the breadth of Cellect’s technology. These latest acknowledgments are significant as they cover the ability to activate stem cells, therefore improving stem cell products and manufacturing processes," commented Dr. Shai Yarkoni, Chief Executive Officer of Cellect. "Our collaboration efforts with companies in Germany, Korea and Israel have each reported similar data using our process in various tissue sources (including fat derived cells). These added protections continue to create barriers to entry and allows us to advance our business development strategies to monetize our innovation."

The Company has previously published third-party data demonstrating improved "stemness" of stem cells. Specifically, it significantly improves both proliferation and functional capabilities of hematopoietic (HSC) and mesenchymal (MSC) stem cells originating from bone marrow, peripheral blood, umbilical cord and adipose tissue.

"We believe the combination of strong IP protection and validated business collaborations support our business model and will enable us to attract external resources to strengthen and expand our opportunities," concluded Dr. Yarkoni.

The patents applications cover an ex vivo method for obtaining an improved population of stem and progenitor cells (SPC) with enhanced engraftment characteristics by activation of TNF family receptors. The Company’s previous patents covered the negative selection exerted by the ApoGraft process and product translated into clinical safety superiority. The latest patent applications describe and protect the positive effect that the same molecules have on stem cells and translates to the efficacy of the transplanted cells and the yields of the manufacturing processes for clinical use).

Including this latest notification, the Company has 65 patent applications worldwide of which 33 are issued/allowed patents.

On January 7, 2020 Clovis Oncology, Inc. (NASDAQ:CLVS) reported its preliminary, unaudited revenues for the fourth quarter and full year ended December 31, 2019 (Press release, Clovis Oncology, JAN 7, 2020, View Source [SID1234552778]). The financial information presented in this news release may be adjusted as a result of completion of customary quarterly review and audit procedures.

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Unaudited preliminary results include:

$38.3-$39.3M in Rubraca product revenues for the fourth quarter of 2019 compared to $37.6M for Q3 2019 and $30.4M for Q4 2018

$142.0-$143.0M in Rubraca product revenues for the FY2019 compared to $95.4M for FY2018

Clovis plans to discuss these results with investors this week at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco.

"We are very pleased with our sales performance in the fourth quarter and the momentum it provides us going into this year," said Patrick J. Mahaffy, CEO and President of Clovis Oncology. "In mid-November 2019, we submitted the supplemental New Drug Application (sNDA) for Rubraca in BRCA1/2-mutant recurrent, metastatic castrate-resistant prostate cancer, and also during the quarter announced the first reported human experience of FAP-2286 and launched Rubraca in England and Italy. With this progress, we are looking forward to an eventful 2020. Key milestones include additional EU country ovarian cancer launches for Rubraca, a potential U.S. Rubraca launch in prostate cancer, initial data for lucitanib combination studies, and a planned IND filing for FAP-2286 in the second half of the year."

Clovis Oncology to Present at 38th Annual J.P. Morgan Healthcare Conference on January 13, 2019

Clovis’ President and CEO, Patrick J. Mahaffy, will present at the 38th Annual J.P. Morgan Healthcare Conference to be held at the Westin St. Francis hotel in San Francisco on Monday, January 13 at 10:00 a.m. PT. A live webcast of the presentation and Q&A session can be accessed through the investor relations section of the Company’s website at clovisoncology.com. Following the live presentations, replays of the webcasts will be available on the Company’s website for 30 days.

Fourth Quarter and Full Year 2019 Financial Results Release Planned for February 24, 2020

The Company plans to report financial results for the fourth quarter and full year ended December 31, 2019 on Monday, February 24, 2020, after the close of the U.S. financial markets. Clovis’ senior management will host a conference call and live audio webcast at 4:30 p.m. ET to discuss the Company’s results in greater detail.

About Rubraca (rucaparib)

Rubraca is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in ovarian cancer as well as several additional solid tumor indications. Studies open for enrollment or under consideration include ovarian, prostate, breast, gastroesophageal, pancreatic, and lung cancers. Clovis holds worldwide rights for Rubraca.

In the United States, Rubraca is approved for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. Rubraca is also approved in the United States for the treatment of adult patients with deleterious BRCA mutation (germline and/or somatic) associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies and selected for therapy based on an FDA-approved companion diagnostic for Rubraca.

In the EU, Rubraca is approved for the maintenance treatment of adults with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy. This expands rucaparib’s indication beyond its initial marketing authorization in the EU granted in May 2018 and with this label expansion, rucaparib is now available to patients regardless of their BRCA mutation status. Rubraca is also approved in the EU for the treatment of adult patients with platinum sensitive, relapsed or progressive, BRCA mutated (germline and/or somatic), high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have been treated with two or more prior lines of platinum-based chemotherapy, and who are unable to tolerate further platinum-based chemotherapy.

Rubraca is an unlicensed medical product outside of the U.S. and the EU.

About Lucitanib

Lucitanib is an oral, potent inhibitor of the tyrosine kinase activity of vascular endothelial growth factor receptors 1 through 3 (VEGFR1-3), platelet-derived growth factor receptors alpha and beta (PDGFRα/b) and fibroblast growth factor receptors 1 through 3 (FGFR1-3). Emerging clinical data support the combination of angiogenesis inhibitors and immunotherapy to increase effectiveness in multiple cancer indications. Angiogenic factors, such as vascular endothelial growth factor (VEGF), are frequently up-regulated in tumors and create an immunosuppressive tumor microenvironment. Use of antiangiogenic drugs reverses this immunosuppression and can augment response to immunotherapy.

Lucitanib is an unlicensed medical product.

About FAP-2286

FAP-2286 is a preclinical candidate discovered by 3B Pharmaceuticals under investigation as a peptide-targeted radionuclide therapy (PTRT) and imaging agent targeting fibroblast activation protein alpha (FAP). FAP is highly expressed in many epithelial cancers, including more than 90 percent of breast, lung, colorectal and pancreatic carcinomas. Clovis will conduct the global clinical trials and holds U.S. and global rights, excluding Europe.

FAP-2286 is an unlicensed medical product.