On September 18, 2018 The Janssen Pharmaceutical Companies of Johnson & Johnson reported that a New Drug Application (NDA) has been submitted to the U.S. Food and Drug Administration (FDA) seeking approval of erdafitinib for the treatment of patients with locally advanced or metastatic urothelial cancer (UC) and certain fibroblast growth factor receptor (FGFR) genetic alterations whose tumors have progressed after prior chemotherapy (Press release, Johnson & Johnson, SEP 18, 2018, View Source [SID1234529481]). Erdafitinib is an investigational, once-daily, oral pan-FGFR inhibitor that received Breakthrough Therapy Designation from the FDA in March 2018.

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!

"Erdafitinib has demonstrated promising results in the treatment of metastatic urothelial cancer, a disease where patients unfortunately have limited treatment options today," said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. "We look forward to working with the FDA in the agency’s review of the application as we believe erdafitinib will provide patients with an important therapeutic option."

The NDA submission is based on data from the BLC2001 (NCT02365597) Phase 2 clinical trial, which evaluated the efficacy and safety of erdafitinib in the treatment of adult patients with locally advanced or metastatic UC, whose tumors have certain FGFR alterations. The primary endpoint of this study was the percentage of participants with objective response, defined as Complete Response or Partial Response based on Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1* criteria. The study results were recently presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2018 Annual Meeting in Chicago (Abstract #4503) and were recognized as a "Best of ASCO (Free ASCO Whitepaper)" selection.

"The erdafitinib FDA submission is an important milestone as we work to bring a new treatment option to patients diagnosed with metastatic urothelial cancer," said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, LLC. "Our organizational focus on areas of high unmet medical need underscores our commitment to advancing transformational science and developing solutions that may prolong and improve patient lives."

*RECIST (version 1.1) refers to Response Evaluation Criteria in Solid Tumors, which is a standard way to measure how well a cancer patient responds to treatment and is based on whether tumors shrink, stay the same, or get bigger.1

For information about Janssen’s pre-approval access program, visit View Source

About Urothelial Cancer

Urothelial cancer, most frequently in the bladder, is the sixth most common type of cancer in the U.S.2 In 2018, an estimated 81,190 new cases of bladder cancer will be diagnosed in the U.S. and an estimated 17,240 bladder cancer deaths will occur.2 The relative five-year survival rate for patients with Stage IV metastatic bladder cancer is currently five percent.3 Patients with metastatic urothelial cancer, who have FGFR genetic alterations, have poor prognoses and a high unmet need based on low response rates and may be resistant to treatment with immune-checkpoint inhibitors.4

About Erdafitinib

Erdafitinib is an investigational, once-daily oral pan-fibroblast growth factor receptor (FGFR) inhibitor being studied in Phase 2 and Phase 3 clinical trials for the treatment of patients with locally advanced or metastatic urothelial cancer.5 FGFRs are a family of receptor tyrosine kinases, which can be activated by genetic alterations in a variety of tumor types, and these alterations may lead to increased tumor cell growth and survival.6 A companion diagnostic to identify patients with FGFR alterations is an integral part of the development program for erdafitinib. In 2008, Janssen entered into an exclusive worldwide license and collaboration agreement with Astex Pharmaceuticals to develop and commercialize erdafitinib.

On September 18, 2018 Actinium Pharmaceuticals, Inc. (NYSE American: ATNM) ("Actinium" or "the Company"), reported that it will unveil a new clinical initiative that expands the Company’s pipeline into the CAR-T space (Press release, Actinium Pharmaceuticals, SEP 18, 2018, View Source [SID1234529483]). CAR-T is a type of cellular therapy that genetically alters a patient’s own T cells to target and kill their cancer cells. Currently, there are 2 approved CAR-T therapies for patients with certain B-cell cancers and over 200 CAR-T candidates in preclinical and clinical development for a wide range of hematologic and solid tumor indications. Actinium’s management team along with Dr. Nirav Shah, Assistant Professor of Medicine, Division or Hematology and Oncology at Froedtert & the Medical College of Wisconsin will discuss the Companies latest pipeline initiative, its value proposition as applicable to CAR-T and the expected development pathway.

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!

Dr. Shah said, "CAR-T is an exciting medical advancement that has demonstrated incredible promise in patients that need better treatment options and outcomes. The Froedtert & the Medical College of Wisconsin was an early adopter of CAR-T becoming one of the first FACT certified medical centers in the nation and commercial CAR-T sites, consistent with its pioneering of other techniques such as stem cell transplantation and immunotherapy. My colleagues and I, at the Froedtert & the Medical College of Wisconsin, are committed to continuing to advance the exciting field of CAR-T to further improve patient outcomes. I am excited by the potential of Actinium’s next generation technology to further advance the field of CAR-T and look forward to providing more details on this potentially disruptive clinical initiative."

In addition to Dr. Shah, Sandesh Seth, Actinium’s Chairman and CEO and Dr. Dale Ludwig, Actinium’s Chief Scientific Officer will be on the call to discuss this new pipeline initiative. Participation details for the conference call and webcast are as follows:

Date: Wednesday, September 26, 2018
Time: 4:15 PM ET
Webcast Registration: View Source
U.S. Participant Dial-in: (718) 865-8336
U.S./Canada Toll Free Dial-in: (855) 427-0225
Conference ID: 4831

"We are excited to be forging a path into CAR-T and are thrilled to be working with Dr. Shah and his colleagues at the Froedtert & Medical College of Wisconsin who have been pioneers in this field. We look forward to the contributions they will make to our next generation technology that we believe has great potential to advance access to CAR-T and patient outcomes’" said, Sandesh Seth, Actinium’s Chairman and CEO. "I am incredibly motivated by this important pipeline expansion which has been enabled by the efforts of our new team and the potential growth opportunities it can provide for Actinium."

On September 18, 2018 OBI Pharma, Inc., a Taiwan biopharma company (TPEx: 4174), reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for OBI-3424 for the Treatment of Acute Lymphoblastic Leukemia (ALL) (Press release, OBI Pharma, SEP 18, 2018, View Source [SID1234529670]). OBI-3424 is a first in class DNA alkylating cancer therapeutic agent targeting aldo-keto reductase 1C3 (AKR1C3) overexpressing cancers.

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!

This is the second FDA orphan drug designation for OBI-3424. In July, 2018, OBI-3424 was granted orphan drug status for the Treatment of Hepatocellular Carcinoma (HCC). A Phase 1/2 study of OBI-3424 in patients with solid tumors, including HCC and castrate-resistant prostate cancer (CRPC), has commenced enrollment at the University of Texas M.D. Anderson Cancer Center.

Amy Huang, General Manager of OBI Pharma, noted, "This additional orphan drug designation for OBI-3424 by the FDA is a significant step in the development of this drug candidate in ALL, including T-ALL, an unmet medical need disease with limited treatment options. We are excited that the FDA has recognized the need to develop novel targeted therapeutic agents such as OBI-3424 in the fight against ALL".

About Acute Lymphoblastic Leukemia (ALL)

Acute Lymphoblastic Leukemia (ALL), also known as Acute Lymphocytic Leukemia, is a rare blood cancer affecting the maturation of B-cell and T-cell lymphoblasts from progenitor cells. The current prevalence of ALL in the US is around 86,462 cases in 2018. The disease affects primarily children, with 60% of cases occurring at age <20 years. The remission rate for pediatric ALL is approximately 90%, with overall survival around 60-70% in recent years. Current treatments for ALL have been less successful in both infant and adult patients, as well as patients with recurrent disease, leading to an unmet medical need for new treatments.

About Orphan Drug Designation (ODD)

The orphan drug designation provides OBI Pharma with potential benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials. The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S.

About OBI-3424

OBI-3424 is a first-in-class novel small-molecule prodrug that selectively targets cancers overexpressing the enzyme aldo-keto reductase 1C3 (AKR1C3), and selectively releases a potent DNA alkylating agent in the presence of the AKR1C3 enzyme. This selective mode of activation distinguishes OBI-3424 from traditional alkylating agents, such as cyclophosphamide and ifosfamide, which are non-selective.

AKR1C3 overexpression has been documented in a number of treatment-resistant and difficult-to-treat cancers including: hepatocellular carcinomas (HCC), castrate-resistant prostate cancer (CRPC), and acute lymphoblastic leukemia (ALL), including T-ALL. AKR1C3 is highly expressed in up to 15 solid and liquid tumors.

OBI Pharma holds worldwide rights for OBI-3424 with the exception of the following countries, whose rights are held by Ascenta Pharma: China, Hong Kong, Macao, Taiwan, Japan, South Korea, Singapore, Malaysia, Thailand, Turkey, and India.

On September 17, 2018 Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to CLR 131 for the treatment of osteosarcoma, a rare pediatric cancer (Press release, Cellectar Biosciences, SEP 17, 2018, View Source [SID1234530175]). CLR 131 is Cellectar’s lead Phospholipid Drug Conjugate (PDC) product candidate.

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!

"CLR 131 has demonstrated promise as an anticancer agent in preclinical and clinical settings, and we are working now to establish its impact on various rare and deadly pediatric cancers," said John Friend, M.D., chief medical officer of Cellectar. "Cellectar is pleased to have the opportunity to work closely with the FDA on our planned Phase 1 trial for these indications and we remain committed to advancing the pediatric programs as rapidly as possible."

Since May 2018 the company has received RPDD for CLR 131 in four pediatric cancers: neuroblastoma, rhabdomyosarcoma, Ewing’s Sarcoma and, most recently, osteosarcoma. Should any of these indications reach approval, the RPDD may enable Cellectar to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive Priority Review for a future NDA or BLA submission, which would reduce the FDA review time from 12 months to six months. Currently, these vouchers can also be transferred or sold to another entity. Over the last 16 months, five priority review vouchers were sold for between $110 million and $150 million each.

The FDA grants Rare Pediatric Disease Designation for diseases that primarily affect children from birth to 18 years old, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

About Osteosarcoma

Osteosarcoma derives from bone forming mesenchymal, or connective tissue, cells and is the most commonly diagnosed primary bone malignancy among children and adolescents. The incidence is about 4.4 cases per 1 million per year in children younger than 24 years [Mirabello 2009]. While there is a 70% cure rate among patients with localized disease, 5-year overall survival rates are approximately 20% for among patients who develop metastatic disease [Saraf 2018]. Additionally, among patients who experience disease progression or recurrence survival for is less than 30% [Chou 2005].

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131 is in a Phase 2 clinical study in R/R MM and a range of B-cell malignancies and a Phase 1b clinical study in patients with R/R MM exploring fractionated dosing. The objective of the multicenter, open-label, Phase 1b dose-escalation study is the characterization of safety and tolerability of CLR 131 in patients with R/R MM. Patients in Cohorts 1-4 received single doses of CLR 131 ranging from 12.5 mCi/m2 to 31.25 mCi/m2. All study doses have been deemed safe and well tolerated by an independent Data Monitoring Committee. The company is currently initiating a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma and is planning a second Phase 1 study in combination with external beam radiation for head and neck cancer.

On September 17, 2018 Coherus BioSciences, Inc. (Nasdaq: CHRS), reported that senior management will be presenting at the 2018 Cantor Global Healthcare Conference being held in New York on Tuesday, October 2, 2018 at 8:00 am ET (Press release, Coherus Biosciences, SEP 17, 2018, View Source;p=RssLanding&cat=news&id=2367664 [SID1234529471]).

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 audio portion of the presentation will be available on the investors page of the Coherus BioSciences website at View Source