Sierra Oncology to Report Chk1 Inhibitor SRA737 Preclinical Data at AACR-NCI-EORTC 2017

On October 16, 2017 Sierra Oncology, Inc. (NASDAQ: SRRA), a clinical stage drug development company focused on advancing next generation DNA Damage Response (DDR) therapeutics for the treatment of patients with cancer, reported that it will report preclinical data supporting the clinical development strategy for its Chk1 inhibitor, SRA737, in a poster to be presented at the upcoming AACR (Free AACR Whitepaper)-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), being held from October 26 – 30, 2017 in Philadelphia, Pennsylvania (Press release, Sierra Oncology, OCT 16, 2017, View Source [SID1234520968]). SRA737 is currently being investigated in two Phase 1 clinical trials in patients with advanced cancers; as a monotherapy, and in combination with low-dose gemcitabine.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Know more, wherever you are:
Latest on AACR (Free AACR Whitepaper)-NCI-EORTC through 1stOncology, book your free 1stOncology demo here.

Title: The Chk1 inhibitor, SRA737, demonstrates chemical synthetic lethality with replication stress-inducing agents, including novel low-dose gemcitabine, in preclinical models of cancer.

Poster #181; Abstract #B181:
Session: Poster Session B; Therapeutic Agents: Small-Molecule Kinase Inhibitors
Date and Time: Sunday, October 29, 2017, 12:30 PM – 4:00 PM
Location: Hall E, Pennsylvania Convention Center
The Poster will be available October 29, 2017 on the company’s website at www.sierraoncology.com.

New Ulcerative Colitis Data for XELJANZ® (tofacitinib) at Upcoming Gastroenterology Congresses

On October 16, 2017 Pfizer Inc. (NYSE:PFE) reported that a total of 10 abstracts on XELJANZ (tofacitinib) in ulcerative colitis (UC) will be presented at the World Congress of Gastroenterology at the American College of Gastroenterology (WCOG at ACG2017) (October 13-18, Orlando, Florida) and the 25th United European Gastroenterology (UEG) Week (October 28–November 1, Barcelona, Spain) (Press release, Pfizer, OCT 16, 2017, View Source [SID1234520967]).
"We are pleased to share this informative data with the gastroenterology community as it deepens our knowledge of tofacitinib in UC and the multi-faceted needs of patients living with the condition," said Michael Corbo, Chief Development Officer, Inflammation & Immunology, Pfizer Global Product Development.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Know more, wherever you are:
Latest on Lung Cancer, book your free 1stOncology demo here.

Pfizer-sponsored research for tofacitinib, which is not currently approved for the treatment of UC, will be presented at the WCOG at ACG2017 and UEG Week. The data include the following presentations:

WCOG at ACG2017 (Times shown in EDT)

Poster Presentations
• Symptomatic Improvement within 3 Days with Tofacitinib Induction Therapy in Patients with Ulcerative Colitis: Results from OCTAVE Induction 1 and 2 [#P418, Sunday, October 15, 2017: 3:30-7:00p.m.]
• Efficacy of Tofacitinib in Patients with Ulcerative Colitis by Prior Tumor Necrosis Factor Inhibitor Treatment Status: Results from OCTAVE Induction and Maintenance Studies [#P449; Sunday, October 15, 2017: 3:30-7:00p.m.]
• Tofacitinib for Maintenance Therapy in Patients with Active Ulcerative Colitis in the Phase 3 OCTAVE Sustain Trial: Results by Local and Central Endoscopic Assessments [#P416; Sunday, October 15, 2017: 3:30-7:00p.m.]
• The Effectiveness of Zoster Vaccine in Patients Subsequently Treated with Tofacitinib [#P2163, Tuesday, October 17, 2017: 10:30a.m.-4:30p.m.]
• Tofacitinib, an Oral Janus Kinase Inhibitor, in the Treatment of Ulcerative Colitis: Open-Label, Long-Term Extension Study [#P2129; Tuesday, October 17, 2017: 10:30a.m.-4:30p.m.]
• An Updated Analysis of In Vitro Cytokine Inhibition Profiles of a Number of Janus Kinase Inhibitors at Clinically Meaningful Concentrations [#P2154; Tuesday, October 17, 2017: 10:30a.m.-4:30p.m.]
• Integrated Safety Analyses of Tofacitinib in Ulcerative Colitis Clinical Trials [#P2146; Tuesday, October 17, 2017: 10:30a.m.-4:30p.m.]
• Pregnancy Outcomes in the Tofacitinib Ulcerative Colitis OCTAVE Studies [#P2155; Tuesday, October 17, 2017: 10:30a.m.-4:30p.m.]

Oral Presentation
• Herpes Zoster Infection in Patients with Ulcerative Colitis Receiving Tofacitinib [#78, Wednesday, October 18, 2017: 9:20-9:30a.m.]

UEG Week (Times shown in UTC)

Poster Presentations
• Comparative Efficacy and Safety of Tofacitinib and Biologics as Induction Therapy for Moderately to Severely Active Ulcerative Colitis: A Systematic Review and Network Meta-Analysis [#P0407, Monday, October 30, 2017: 9:00a.m.-5:00p.m.]
• Pregnancy Outcomes in the Tofacitinib Ulcerative Colitis OCTAVE Studies [#P0394; Monday, October 30, 2017: 9:00a.m.-5:00p.m.] Encore presentation from the WCOG at ACG2017

Oral Presentations
• Tofacitinib, an Oral Janus Kinase Inhibitor, in the Treatment of Ulcerative Colitis: Open-Label, Long-Term Extension Study [#OP095; Monday, October 30, 2017: 3:45-5:15p.m.] Encore presentation from the WCOG at ACG2017
• Tofacitinib for Maintenance Therapy in Patients with Active Ulcerative Colitis in the Phase 3 OCTAVE Sustain Trial: Results by Local and Central Endoscopic Assessments [#OP059; Monday, October 30, 2017: 2:00-3:30p.m.] Encore presentation from the WCOG at ACG2017
• An Updated Analysis of In Vitro Cytokine Inhibition Profiles of a Number of Janus Kinase Inhibitors at Clinically Meaningful Concentrations [#OP222; Tuesday, October 31, 2017: 10:30a.m.-12:00p.m.] Encore presentation from the WCOG at ACG2017
• Integrated Safety Analyses of Tofacitinib in Ulcerative Colitis Clinical Trials [#OP353; Wednesday, November 1, 2017: 10:30a.m.-12:00p.m.] Encore presentation from the WCOG at ACG2017

About Ulcerative Colitis

UC is a chronic, debilitating and often misunderstood inflammatory bowel disease that affects millions of people worldwide. 1,2 Symptoms of UC can include chronic diarrhea with blood and mucus, abdominal pain and cramping, fever and weight loss. 3a,4 While the exact cause of UC is unknown, it is believed to be the result of complex interactions between multiple factors that include genetic predisposition and an exaggerated immune response to a microbial trigger.5 UC can have a significant effect on work, family and social activities. 6 Despite receiving treatment, half of patients continue to experience symptoms.7 Under these circumstances, surgery to remove the colon (colectomy), may be considered for some patients.3b

About Tofacitinib
Tofacitinib is a Janus kinase (JAK) inhibitor. Applications for tofacitinib for the treatment of moderately to severely active UC are currently under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). It is not currently approved for the treatment of UC.
As the developer of tofacitinib, Pfizer is a leader in JAK science and is committed to enhancing understanding of tofacitinib through robust clinical development programs in the treatment of immune-mediated inflammatory conditions.

INDICATION
Rheumatoid Arthritis
• XELJANZ/XELJANZ XR (tofacitinib) is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate. It may be used as monotherapy or in combination with methotrexate or other nonbiologic disease-modifying antirheumatic drugs (DMARDs).
• Limitations of Use: Use of XELJANZ/XELJANZ XR in combination with biologic DMARDs or with potent immunosuppressants such as azathioprine and cyclosporine is not recommended.

IMPORTANT SAFETY INFORMATION
BOXED WARNING: SERIOUS INFECTIONS AND MALIGNANCY
SERIOUS INFECTIONS

Patients treated with XELJANZ/XELJANZ XR are at increased risk for developing serious infections that may lead to hospitalization or death. Most patients who developed these infections were taking concomitant immunosuppressants, such as methotrexate or corticosteroids.
If a serious infection develops, interrupt XELJANZ/XELJANZ XR until the infection is controlled.
Reported infections include:
• Active tuberculosis, which may present with pulmonary or extrapulmonary disease. Patients should be tested for latent tuberculosis before XELJANZ/XELJANZ XR use and during therapy. Treatment for latent infection should be initiated prior to XELJANZ/XELJANZ XR use.
• Invasive fungal infections, including cryptococcosis and pneumocystosis. Patients with invasive fungal infections may present with disseminated, rather than localized, disease.
• Bacterial, viral, and other infections due to opportunistic pathogens.
The risks and benefits of treatment with XELJANZ/XELJANZ XR should be carefully considered prior to initiating therapy in patients with chronic or recurrent infection.
Patients should be closely monitored for the development of signs and symptoms of infection during and after treatment with XELJANZ/XELJANZ XR, including the possible development of tuberculosis in patients who tested negative for latent tuberculosis infection prior to initiating therapy.

MALIGNANCIES
Lymphoma and other malignancies have been observed in patients treated with XELJANZ. Epstein Barr Virus-associated post-transplant lymphoproliferative disorder has been observed at an increased rate in renal transplant patients treated with XELJANZ and concomitant immunosuppressive medications.

WARNINGS AND PRECAUTIONS
SERIOUS INFECTIONS

The most common serious infections reported with XELJANZ included pneumonia, cellulitis, herpes zoster, urinary tract infection, diverticulitis, and appendicitis. Avoid use of XELJANZ/XELJANZ XR in patients with an active, serious infection, including localized infections. Consider the risks and benefits of treatment before initiating XELJANZ/XELJANZ XR in patients:
• with chronic or recurrent infection;
• who have been exposed to tuberculosis (TB);
• with a history of a serious or an opportunistic infection;
• who have lived or traveled in areas of endemic TB or mycoses; or
• with underlying conditions that may predispose them to infection.
Patients should be closely monitored for the development of signs and symptoms of infection during and after treatment with XELJANZ/XELJANZ XR. XELJANZ/XELJANZ XR should be interrupted if a patient develops a serious infection, an opportunistic infection, or sepsis.

Caution is also recommended in patients with a history of chronic lung disease, or in those who develop interstitial lung disease, as they may be more prone to infection.
Risk of infection may be higher with increasing degrees of lymphopenia and consideration should be given to lymphocyte counts when assessing individual patient risk of infection.

Tuberculosis
Evaluate and test patients for latent or active infection prior to and per applicable guidelines during administration of XELJANZ/XELJANZ XR. Consider anti-TB therapy prior to administration of XELJANZ/XELJANZ XR in patients with a past history of latent or active TB in whom an adequate course of treatment cannot be confirmed, and for patients with a negative test for latent TB but who have risk factors for TB infection. Treat patients with latent TB with standard therapy before administering XELJANZ/XELJANZ XR.

Viral Reactivation
Viral reactivation, including cases of herpes virus reactivation (e.g., herpes zoster), was observed in clinical studies with XELJANZ. Screening for viral hepatitis should be performed in accordance with clinical guidelines before starting therapy with XELJANZ/XELJANZ XR. The risk of herpes zoster is increased in patients treated with XELJANZ/XELJANZ XR and appears to be higher in patients treated with XELJANZ in Japan and Korea.
MALIGNANCY and LYMPHOPROLIFERATIVE DISORDERS

Consider the risks and benefits of XELJANZ/XELJANZ XR treatment prior to initiating therapy in patients with a known malignancy other than a successfully treated non-melanoma skin cancer (NMSC) or when considering continuing XELJANZ/XELJANZ XR in patients who develop a malignancy.

In the 7 controlled rheumatoid arthritis clinical studies, 11 solid cancers and 1 lymphoma were diagnosed in 3328 patients receiving XELJANZ with or without DMARD, compared to 0 solid cancers and 0 lymphomas in 809 patients in the placebo with or without DMARD group during the first 12 months of exposure. Lymphomas and solid cancers have also been observed in the long-term extension studies in rheumatoid arthritis patients treated with XELJANZ.
In Phase 2B controlled dose-ranging trials in de-novo renal transplant patients, all of whom received induction therapy with basiliximab, high-dose corticosteroids, and mycophenolic acid products, Epstein Barr Virus-associated post-transplant lymphoproliferative disorder was observed in 5 out of 218 patients treated with XELJANZ (2.3%) compared to 0 out of 111 patients treated with cyclosporine.
Other malignancies were observed in clinical studies and the post-marketing setting including, but not limited to, lung cancer, breast cancer, melanoma, prostate cancer, and pancreatic cancer.

Non-Melanoma Skin Cancer
Non-melanoma skin cancers (NMSCs) have been reported in patients treated with XELJANZ. Periodic skin examination is recommended for patients who are at increased risk for skin cancer.
GASTROINTESTINAL PERFORATIONS
Gastrointestinal perforations have been reported in XELJANZ rheumatoid arthritis clinical trials, although the role of JAK inhibition is not known. XELJANZ/XELJANZ XR should be used with caution in patients who may be at increased risk for gastrointestinal perforation (e.g., patients with a history of diverticulitis).
LABORATORY ABNORMALITIES

Lymphocyte Abnormalities
Treatment with XELJANZ was associated with initial lymphocytosis at 1 month of exposure followed by a gradual decrease in mean lymphocyte counts of approximately 10% during 12 months of therapy. Counts less than 500 cells/mm3 were associated with an increased incidence of treated and serious infections. Avoid initiation of XELJANZ/XELJANZ XR treatment in patients with a count less than 500 cells/mm3. In patients who develop a confirmed absolute lymphocyte count less than 500 cells/mm3, treatment with XELJANZ/XELJANZ XR is not recommended. Monitor lymphocyte counts at baseline and every 3 months thereafter.

Neutropenia
Treatment with XELJANZ was associated with an increased incidence of neutropenia (less than 2000 cells/mm3) compared to placebo. Avoid initiation of XELJANZ/XELJANZ XR treatment in patients with an ANC less than 1000 cells/mm3. For patients who develop a persistent ANC of 500-1000 cells/mm3, interrupt XELJANZ/XELJANZ XR dosing until ANC is greater than or equal to 1000 cells/mm3. In patients who develop an ANC less than 500 cells/mm3, treatment with XELJANZ/XELJANZ XR is not recommended. Monitor neutrophil counts at baseline and after 4-8 weeks of treatment and every 3 months thereafter.

Anemia
Avoid initiation of XELJANZ/XELJANZ XR treatment in patients with a hemoglobin level less than 9 g/dL. Treatment with XELJANZ/XELJANZ XR should be interrupted in patients who develop hemoglobin levels less than 8 g/dL or whose hemoglobin level drops greater than 2 g/dL on treatment. Monitor hemoglobin at baseline and after 4-8 weeks of treatment and every 3 months thereafter.

Liver Enzyme Elevations
Treatment with XELJANZ was associated with an increased incidence of liver enzyme elevation compared to placebo. Most of these abnormalities occurred in studies with background DMARD (primarily methotrexate) therapy.
Routine monitoring of liver tests and prompt investigation of the causes of liver enzyme elevations is recommended to identify potential cases of drug-induced liver injury. If drug-induced liver injury is suspected, the administration of XELJANZ/XELJANZ XR should be interrupted until this diagnosis has been excluded.

Lipid Elevations
Treatment with XELJANZ was associated with increases in lipid parameters, including total cholesterol, low-density lipoprotein (LDL) cholesterol, and high-density lipoprotein (HDL) cholesterol. Maximum effects were generally observed within 6 weeks.
Assess lipid parameters approximately 4-8 weeks following initiation of XELJANZ/XELJANZ XR therapy, and manage patients according to clinical guidelines for the management of hyperlipidemia.

VACCINATIONS
Avoid use of live vaccines concurrently with XELJANZ/XELJANZ XR. The interval between live vaccinations and initiation of tofacitinib therapy should be in accordance with current vaccination guidelines regarding immunosuppressive agents. A varicella virus-naïve patient experienced dissemination of the vaccine strain of varicella zoster virus 16 days after vaccination with live attenuated virus vaccine which was 2 days after 5 mg twice daily treatment with tofacitinib. The patient recovered after discontinuation of tofacitinib and treatment with antiviral medication. Update immunizations in agreement with current immunization guidelines prior to initiating XELJANZ/XELJANZ XR therapy.

GENERAL
Specific to XELJANZ XR
Caution should be used when administering XELJANZ XR to patients with pre-existing severe gastrointestinal narrowing. There have been rare reports of obstructive symptoms in patients with known strictures in association with the ingestion of other drugs utilizing a non-deformable extended release formulation.

HEPATIC and RENAL IMPAIRMENT
Use of XELJANZ/XELJANZ XR in patients with severe hepatic impairment is not recommended. The recommended dose in patients with moderate hepatic impairment or with moderate or severe renal impairment is XELJANZ 5 mg once daily.

ADVERSE REACTIONS
The most common serious adverse reactions were serious infections. The most commonly reported adverse reactions during the first 3 months in controlled clinical trials with XELJANZ 5 mg twice daily and placebo, respectively, (occurring in greater than or equal to 2% of patients treated with XELJANZ with or without DMARDs) were upper respiratory tract infections (4.5%, 3.3%), headache (4.3%, 2.1%), diarrhea (4.0%, 2.3%), and nasopharyngitis (3.8%, 2.8%).

USE IN PREGNANCY
There are no adequate and well-controlled studies in pregnant women and the estimated background risks of major birth defects and miscarriage for the indicated population is unknown. Based on animal studies, tofacitinib has the potential to affect a developing fetus. Women of reproductive potential should be advised to use effective contraception.

FENNEC ANNOUNCES POSITIVE RESULTS FROM PHASE 3 SIOPEL 6 STUDY ON PEDMARKTM (sodium thiosulfate) PRESENTED AT THE 49TH CONGRESS OF THE INTERNATIONAL SOCIETY OF PEDIATRIC ONCOLOGY (SIOP) 2017 MEETING

On October 16, 2017 Fennec Pharmaceuticals Inc. (NASDAQ: FENC, TSX: FRX), a specialty pharmaceutical company focused on the development of PEDMARKTM (a unique formulation of sodium thiosulfate (STS)) for the prevention of platinum-induced ototoxicity in pediatric patients, reported data from its Phase 3 SIOPEL 6 study presented during the late breaker session on Saturday, October 14, 2017 at SIOP 2017 in Washington, DC (Press release, Fennec Pharmaceuticals, OCT 16, 2017, View Source [SID1234520965]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Know more, wherever you are:
Latest on Pediatric Cancer Drug Development, book your free 1stOncology demo here.

Top Line Efficacy Data

The SIOPEL 6 study met its primary endpoint. The study demonstrated that the addition of STS significantly reduces the incidence of cisplatin-induced hearing loss without any evidence of tumour protection. Among the 99 evaluable patients, hearing loss occurred in 30/45=67% treated with Cisplatin (Cis) alone and in 20/54=37.0% treated with Cis+STS, corresponding to a relative risk of 0.56(P=0.0033).

Fennec plans to pursue regulatory approval for PEDMARKTM based on the data from the SIOPEL 6 study along with the proof of principle data from COG ACCL0431. STS has received Orphan Drug Designation in the US in this setting and plans to pursue European Market Exclusivity for Pediatric Use upon approval.

"I am absolutely delighted that after 30 years of research we have found a safe way to reduce ototoxicity in children receiving platinum containing chemotherapy," said Penelope Brock, M.D., PhD, International Chair of SIOPEL. "This means that children who are cured from cancer after receiving platinum treatment can look forward to a normal healthy life, fully integrated into society. I believe this marks a new standard of care in pediatric oncology."
The Company also reported top-line data for secondary endpoints Event Free Survival (EFS) and Overall Survival (OS). The combination of Cis+STS was generally well tolerated. With a follow up of 52 months, 3yr EFS is Cis 78.8% and Cis+STS 82.1%; 3yr OS is Cis 92.3% and Cis+STS 98.2%.

"We are very pleased with the results of this study," stated Rosty Raykov, CEO of Fennec. " We would like to thank all the patients and their families who participated in this trial, physicians, the entire SIOPEL 6 team, and Dr. Neuwelt and his research team at OHSU. We believe that if approved PEDMARK would be an important therapy for patients and caregivers where currently there are no treatment options."

Safety and Tolerability

In the study, the results presented showed that treatment was well tolerated and acute toxicity similar and expected between arms. The table below presents the toxicities of the two arms:

Adverse event Grade CIS CIS+STS
N % N %
Febrile neutropenia 3 7 13.5 5 8.8
4 - - - -
Infection 3 5 9.6 6 10.5
4 - - - -
Hypomagnesemia 3 1 1.9 1 1.8
4 - - - -
Hypernatremia 3 - - 1 1.8
4 - - - -
Vomiting 3 1 1.9 3 5.3
4 - - - -
Nausea 3 3 5.8 2 3.5
4 - - - -

SIOP 2017 Presentation

Fennec will provide access to the recording of SIOP 2017 late breaker presentation on the Company’s website.

To access the archived recording, visit the Fennec website at www.fennecpharma.com.

SIOPEL 6

SIOPEL 6 is a multi-centre open label randomized phase 3 study evaluating the efficacy of STS in reducing ototoxicity in patients receiving cisplatin monotherapy for standard risk hepatoblastoma. From the beginning of 2007 to the end 2014, 52 sites from 11 countries enrolled 113 evaluable patients. The study is closed to recruitment and all protocol pre-specified IDMC safety reviews are now complete. The primary efficacy hearing endpoint analysis can be performed once patients have reached 3.5 years of age and an audiometry test can be carried out. The SIOPEL 6 study trial was designed with 80% power and a 5% significance level to detect an absolute 25% reduction in the rate of Brock grade ≥1 hearing loss with a chi-square test, from a 60% hearing loss in Cis alone arm to a 35% hearing loss in Cis+STS arm. The primary endpoint is the rate of Brock grade ≥ 1 hearing loss determined after the end of treatment at the age of ≥3.5 years by pure tone audiometry.

About PEDMARKTM (sodium thiosulfate/STS)

Cisplatin and other platinum compounds are essential chemotherapeutic components for many pediatric malignancies. Unfortunately, platinum-based therapies cause ototoxicity in many patients, and are particularly harmful to the survivors of pediatric cancer.

In the U.S. and Europe there is estimated that over 7,000 children are diagnosed with local cancers that may receive platinum-based chemotherapy. Localized cancers that receive platinum agents may have overall survival rates of greater than 80% further emphasizing the quality of life after treatment. The incidence of hearing loss in these children depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

STS has been studied by cooperative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, The Clinical Oncology Group Protocol ACCL0431 and SIOPEL 6. Both studies are closed to recruitment. The COG ACCL0431 protocol enrolled one of five childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors. COG ACCL0431 final results were published in the Lancet Oncology.

In May 2017, Fennec announced the launch of a Named Patient Program in Europe. European based Healthcare Professionals can obtain details about STS Named Patient Program by emailing [email protected].

Ipsen announces that phase 3 CELESTIAL trial of cabozantinib meets primary endpoint of overall survival in patients with advanced hepatocellular carcinoma

On 16 October, 2017 Ipsen (Euronext: IPN; ADR: IPSEY) and its partner Exelixis (NASDAQ: EXEL) reported that its global phase 3 CELESTIAL trial met its primary endpoint of overall survival (OS), with cabozantinib providing a statistically significant and clinically meaningful improvement in median OS compared to placebo in patients with advanced hepatocellular carcinoma (HCC) (Press release, Ipsen, OCT 16, 2017, View Source [SID1234520945]). The independent data monitoring committee for the study recommended that the trial should be stopped for efficacy following review of the second planned interim analysis. CELESTIAL is a randomized, global phase 3 trial of cabozantinib versus placebo in patients with advanced HCC who have been previously treated with sorafenib. The safety data in the study were consistent with the established profile of cabozantinib.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In line with and in collaboration with our partner Exelixis, Ipsen expects to file in the first half of 2018 a variation of the initial application to the EMA and other relevant regulatory agencies and to evaluate potential next steps in the development strategy for cabozantinib outside the United States and Japan as a treatment for advanced HCC in patients who have been previously treated. Detailed results from CELESTIAL will be submitted for presentation at a future medical conference.

Alexandre Lebeaut, MD, Executive Vice-President, R&D, Chief Scientific Officer, Ipsen, said: "Liver cancer is one of the leading causes of cancer deaths worldwide and more effective treatment options are urgently needed. We are pleased to report that in the CELESTIAL clinical study cabozantinib has been shown to provide a survival benefit and therefore has the potential to bring a new oral systemic treatment to previously treated patients with advanced liver cancer. "

About the CELESTIAL Study

CELESTIAL is a randomized, double-blind, placebo-controlled study of cabozantinib in patients with advanced HCC conducted at more than 100 sites globally in 19 countries. The trial was designed to enroll 760 patients with advanced HCC who previously received sorafenib and may have received up to two prior systemic cancer therapies for HCC and had adequate liver function. Enrollment of the trial was completed in September 2017, and 773 patients were ultimately randomized. Patients were randomized 2:1 to receive 60 mg of cabozantinib once daily or placebo and were stratified based on etiology of the disease (hepatitis C, hepatitis B or other), geographic region (Asia versus other regions) and presence of extrahepatic spread and/or macrovascular invasion (yes or no). No cross-over was allowed between the study arms.

The primary endpoint for the trial is OS, and secondary endpoints include objective response rate and progression-free survival. Exploratory endpoints include patient-reported outcomes, biomarkers and safety.

Based on available clinical trial data from various published trials conducted in the second-line setting of advanced HCC, the CELESTIAL trial statistics for the primary endpoint of OS assumed a median OS of 8.2 months for the placebo arm. A total of 621 events provide the study with 90 percent power to detect a 32 percent increase in median OS (HR = 0.76) at the final analysis. Two interim analyses were planned and conducted at 50 percent and 75 percent of the planned 621 events.

About HCC

Hepatocellular Carcinoma (HCC) is the most common form of liver cancer in adults.1 The disease originates in cells called hepatocytes found in the liver. With approximately 800’000 new cases diagnosed each year, HCC is the sixth most common cancer and the second-leading cause of cancer deaths worldwide.2,3 According to the GLOBOCAN data, it is estimated that across the European Union (EU-28) nearly 60’000 new patients will be diagnosed with liver cancer in 2020.4 Without treatment, patients with the disease in advanced stage usually survive between 4 and 8 months.5

About CABOMETYX (cabozantinib)

Cabometyx is an oral small molecule inhibitor of receptors, including VEGFR, MET, AXL and RET. In preclinical models, cabozantinib has been shown to inhibit the activity of these receptors, which are involved in normal cellular function and pathologic processes such as tumor angiogenesis, invasiveness, metastasis and drug resistance.

In February of 2016, Exelixis and Ipsen jointly announced an exclusive licensing agreement for the commercialization and further development of cabozantinib indications outside of the United States, Canada and Japan. This agreement was amended in December of 2016 to include commercialization rights for Ipsen in Canada. On April 25, 2016, the FDA approved Cabometyx tablets for the treatment of patients with advanced RCC who have received prior anti-angiogenic therapy and on September 9, 2016, the European Commission approved Cabometyx tablets for the treatment of advanced RCC in adults who have received prior vascular endothelial growth factor (VEGF)-targeted therapy in the European Union, Norway and Iceland. Cabometyx is available in 20 mg, 40 mg or 60 mg doses. The recommended dose is 60 mg orally, once daily.

Ipsen also submitted to European Medicines Agency (EMA) the regulatory dossier for cabozantinib as a treatment for first-line advanced RCC in the European Union on August 28, 2017; on September 8, 2017, Ipsen announced that the EMA validated the application.

Cabozantinib is not approved for the treatment of advanced hepatocellular carcinoma.

Atossa Genetics Announces that Rutgers, The State University at New Jersey, Plans Study Utilizing Atossa’s Microcatheter Technology

On October 16, 2017 Atossa Genetics (NASDAQ:ATOS), a clinical-stage pharmaceutical company developing novel therapeutics and delivery methods for breast cancer and other breast conditions, reported that the Ernest Mario School of Pharmacy at Rutgers, The State University at New Jersey, plans to conduct a study utilizing Atossa’s intraductal microcatheter technology (Press release, Atossa Genetics, OCT 16, 2017, View Source [SID1234520936]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Rutgers researchers believe that directly administering drugs into the breast duct where breast cancer grows, by inserting microcatheters into the nipple, is a better alternative than systemic administration, because the drugs will be directly delivered to the tissue. The Rutgers program uses a unique directed delivery system comprised of nanoscale pharmaceutical carriers loaded with single drugs. The long-term goal of the research program is to develop a locally administered drug delivery system that selectively targets and delivers pathway-specific targeting therapeutics to eliminate breast cancer cells and cancer stem-like cells while sparing normal breast cells.

"We are encouraged that a leading research institution like Rutgers recognizes the potential merit of our microcatheter technology. Atossa fully supports additional research utilizing our patented microcatheter technology," stated Steven Quay, MD, PhD, Atossa CEO and President.

The Rutgers program is in the research and development phase and has not been approved by the FDA or any other regulatory body. Studies demonstrating safety and efficacy, among other things, and regulatory approvals will be required before commercialization.