Phase 3 Registration Trial for Non-Muscle Invasive Bladder Cancer Achieves 42% Complete Response Rate at Three Months in Carcinoma in Situ Patients

On May 21, 2018 Sesen Bio, Inc. (NASDAQ: SESN), a late-stage clinical company advancing next-generation antibody-drug conjugate (ADC) therapies for the treatment of cancer, reported positive, three-month data from its ongoing Phase 3 VISTA Trial of Vicinium for the treatment of patients with high-grade non-muscle invasive bladder cancer (NMIBC) who have been previously treated with bacillus Calmette-Guérin (BCG) (Press release, Eleven Biotherapeutics, MAY 21, 2018, View Source [SID1234526819]). The efficacy data being reported are based on three-month follow-ups from 111 patients with high-grade NMIBC that is either carcinoma in situ (CIS), which is cancer found on the inner lining of the bladder that has not spread into muscle or other tissue, with or without papillary disease, or from patients with papillary disease without CIS, which is cancer that has grown from the bladder lining out into the bladder, but has not spread into muscle or other tissue.

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!

"High-grade NMIBC is a devastating cancer that typically occurs later in life and for which treatment options are limited. Over the last 30 years, our industry has seen little innovation for the treatment of this prevalent cancer. The current standard-of-care, BCG, works in many patients, but many will also eventually relapse. For those patients who relapse or who don’t respond at all, the standard alternative is radical cystectomy. In a cystectomy, the bladder is removed along with surrounding lymph nodes and other organs that contain cancer. I am very encouraged by both the safety and these three-month efficacy data with Vicinium, and I look forward to continuing to work with the Sesen Bio team to help bring forward this potential treatment as a safe and effective option for my patients," said Rian Dickstein, M.D. F.A.C.S., chief of urology, University of Maryland Baltimore Washington Medical Center; medical director of GU oncology, Tate Cancer Center at The University of Maryland Baltimore Washington Medical Center​; clinical assistant professor, Department of Surgery, University of Maryland School of Medicine; director, bladder cancer program, Chesapeake Urology; and an investigator in the VISTA Trial.

"The VISTA Trial three-month data are encouraging for our company and the patients with high-grade NMIBC who have been underserved for many years," said Stephen Hurly, president and chief executive officer of Sesen Bio. "We have made tremendous progress over the last several years to get us to where we are today, and I am proud of what our team has accomplished. Our new name is a reflection of the journey we’ve taken to get to this point and represents our

1

sesnauadataprvfinalimage1.jpg

mission of improving lives. With 12-month data expected by mid-2019, we are continuing to advance Vicinium to assess its full potential in treating this devastating cancer."

VISTA Trial Design and Patient Cohorts
The Phase 3 VISTA Trial completed recruitment in March 2018 with a total of 133 patients with high-grade NMIBC that is either CIS or papillary with or without CIS, who have been previously treated with BCG. The primary endpoint of the trial is the complete response rate in patients with CIS with or without papillary disease.

The clinical trial includes three patient cohorts based on histology and time to recurrence after adequate BCG:
•
Cohort 1 (n=87): patients with CIS with or without papillary disease whose cancer recurred within six months of their last course of BCG treatment.
•
Cohort 2 (n=6): patients with CIS with or without papillary disease whose cancer recurred after six months, but before 11 months, after their last course of BCG treatment.
•
Cohort 3 (n=40): patients with papillary disease without CIS whose cancer recurred within six months of their last course of BCG treatment.
Three-Month Efficacy Results: CIS Patients
In cohort 1, 72 patients were evaluable for three-month data as of the April 20, 2018 data cut-off date. In these patients, treatment with Vicinium demonstrated a complete response rate of 39 percent. In evaluable patients in cohort 2 (n=5), treatment with Vicinium demonstrated a complete response rate of 80 percent. In an analysis assessing pooled CIS patients from cohorts 1 and 2 (n=77), based on final U.S. Food and Drug Administration guidance on treatment of BCG-unresponsive CIS NMIBC patients (defined as patients with recurrent CIS within 12 months of adequate BCG therapy), Vicinium treatment resulted in a complete response rate of 42 percent at three months.

Three-Month Efficacy Results: Papillary Patients
Patients with papillary disease without CIS were enrolled in cohort 3, but are not included in the primary endpoint assessment. At screening, all of these patients underwent mandatory resection of their tumors and upon starting treatment, were deemed to have no visible evidence of disease. As such, in this patient population, rates of disease recurrence and time to disease recurrence are standard criteria to evaluate response. In these evaluable patients (n=34), treatment with Vicinium demonstrated a 68 percent recurrence-free rate at three months.

Preliminary Safety Results
To date, Vicinium has been well-tolerated in the VISTA Trial. In treated patients across cohorts (n=129), 72 percent of all adverse events were Grade 1 or 2. The most commonly reported treatment-emergent adverse events (all grades) were urinary tract infection (29%), dysuria (19%), hematuria (16%), pollakiuria (12%), diarrhea (10%), fatigue (10%), micronutrition urgency (9%), nausea (8%) and increased lipase (8%, all asymptomatic). Of the treatment-related adverse events, four percent were Grade 3 or 4, with no Grade 5 treatment-related adverse events. Four treatment-related serious adverse events were reported, including acute kidney injury or renal failure and cholestatic hepatitis.

"The positive, three-month data from the VISTA Trial support Vicinium’s potential to offer a new and completely different treatment option for patients who need it," said Donald L. Lamm, M.D., BCG Oncology, P.C., Phoenix. "In contrast to the many other advances in cancer therapy, treatment for high-grade NMIBC has suffered from little innovation, and Vicinium represents a unique approach with a targeted protein, an entirely new mechanism for treating this disease. I believe this new treatment approach, which has demonstrated a 42 percent, three-month complete response rate in patients who have failed to respond to BCG and good tolerability, gives these patients new hope for beating bladder cancer without life-changing major surgery. High-grade NMIBC patients have a high chance of losing their bladder after having gone through unreliable and often difficult treatments. I believe that Vicinium, a first-in-class, innovative therapy, may change that in the future."

Conference Call Information
To participate in the conference call, please dial (844) 831-3025 (domestic) or
(315) 625-6887 (international) and refer to conference ID 4453267. The webcast can be accessed in the Investor Relations section of the company’s website at www.sesenbio.com. The replay of the webcast will be available in the investor section of the company’s website at www.sesenbio.com for 60 days following the call.

About the VISTA Clinical Trial
The VISTA Trial is an open-label, multicenter, single-arm Phase 3 clinical trial evaluating the efficacy and tolerability of Vicinium in patients with high-grade non-muscle invasive bladder cancer (NMIBC) that is carcinoma in situ (CIS), which is cancer found on the inner lining of the bladder that has not spread into muscle or other tissue) and/or papillary, which is cancer that has grown from the bladder lining out into the bladder but has not spread into muscle or other tissue, who have been previously treated with bacillus Calmette-Guérin (BCG). The primary endpoint of the trial is the complete response rate in patients with CIS with or without papillary disease. Patients in the trial receive locally administered Vicinium twice a week for six weeks, followed by once-weekly treatment for another six weeks, then treatment every other week for up to two years. Twelve-month data are anticipated in mid-2019. To learn more about the Phase 3 VISTA Trial, please visit www.clinicaltrials.gov and search the identifier NCT02449239.

About Vicinium
Vicinium, also known as VB4-845, is Sesen Bio’s lead product candidate and is a next-generation antibody-drug conjugate (ADC), developed using the company’s proprietary Targeted Protein Therapeutics platform, for the treatment of high-grade non-muscle invasive bladder cancer (NMIBC). Vicinium is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A (ETA). Vicinium is constructed with a stable, genetically engineered peptide linker to ensure the payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical trials conducted by Sesen Bio, EpCAM has been shown to be overexpressed in NMIBC cells with minimal to no EpCAM expression

observed on normal bladder cells. Sesen Bio is currently conducting the Phase 3 VISTA Trial, designed to support the registration of Vicinium for the treatment of high-grade NMIBC in patients who have previously received two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Twelve-month data from the trial are anticipated in mid-2019. Additionally, Sesen Bio believes that Vicinium’s cancer cell-killing properties promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. The activity of Vicinium in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.

About Non-Muscle Invasive Bladder Cancer
Bladder cancer is the sixth most commonly diagnosed cancer in the United States, and approximately 80 percent of patients have non-muscle invasive bladder cancer (NMIBC). In NMIBC, cancer cells are in the lining of the bladder or have grown into the lumen of the bladder but have not spread into muscle or other tissue. NMIBC primarily affects men and is associated with carcinogen exposure. Initial treatment includes surgical resection; however, there is a high rate of recurrence and more than 60 percent of all patients diagnosed with NMIBC will receive bacillus Calmette-Guérin (BCG) immunotherapy. While BCG is effective in many patients, challenges with tolerability have been observed and many patients will experience recurrence of disease. If BCG is not effective or a patient can longer receive BCG, the recommended option for treatment is radical cystectomy, the complete removal of the bladder.

Regeneron Announces Upcoming Investor Conference Presentation

On May 18, 2018 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported it will webcast its management presentation at the Goldman Sachs 39th Annual Global Healthcare Conference at 8:40 a.m. Pacific Time (11:40 a.m. Eastern Time) on Tuesday, June 12, 2018 (Press release, Regeneron, MAY 18, 2018, View Source [SID1234526799]).

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 session may be accessed from the "Investors & Media" page of Regeneron’s website at View Source A replay of the webcast will be archived on the Company’s website and will be available for 30 days

Rocket Pharmaceuticals Presents Updated Data from Phase 1/2 Gene Therapy Trial of RP-L102 in Patients with Fanconi Anemia at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

On May 18, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported the presentation of updated data from the ongoing Phase 1/2 clinical trial of RP-L102, the Company’s lead lentiviral vector (LVV)-based gene therapy, for Fanconi Anemia (FA) (Press release, Rocket Pharmaceuticals, MAY 18, 2018, View Source;p=RssLanding&cat=news&id=2349899 [SID1234526803]). The data were highlighted today in an oral presentation during the distinguished Presidential Symposium at the ASGCT (Free ASGCT Whitepaper) 2018 Annual Meeting, by Dr. Juan Bueren, Head of the Hematopoietic Innovative Therapies Division at the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain / CIBER-Rare Diseases / IIS-Fundación Jiménez Díaz (FJD), and program principal investigator of the RP-L102 trial.

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!

"Several important observations are emerging from our ongoing Phase 1/2 trial in FA. First, even without myeloablative conditioning, there are increasing levels of bone marrow engraftment following administration of RP-L102. Second, the improvement of chromosomal stability in corrected FA cells indicates that RP-L102 is reversing the FA phenotype. Third, the natural progression of bone marrow failure in these patients is reversed. In fact, the bone marrow cells of the two patients who received higher doses demonstrate conversion to a somatic mosaic status that is sustained over the course of several months. Finally, the progressive increases of corrected, versus non-corrected, peripheral blood leukocytes indicate that RP-L102 is restoring functionality of bone marrow hematopoietic stem cells. This translates to a stabilization in peripheral blood cell counts which would otherwise continue to decline in the absence of treatment. Based on these encouraging results, I believe that RP-L102 has the potential to be a transformative and minimally toxic prevention of bone marrow failure for FA," said Dr. Bueren.

This Phase 1/2 study is an ongoing, open-label, single-center study designed to evaluate the safety and efficacy of RP-L102 in FA type A without the use of myeloablative conditioning. Julian Sevilla, M.D., Ph.D., of the hospital of Niño Jesús in Madrid, is the clinical trial principal investigator. Five patients have been treated to date. The first four patients have been followed for 12-24 months, and a fifth patient, treated with transduction-enhanced RP-L102, has been followed for two months.

Key efficacy measurements include:

Genetic correction of bone marrow cells (engraftment): measured by peripheral blood vector copy number (VCN)
Functional and phenotypic correction of bone marrow cells: measured by resistance to mitomycin-C (MMC)
Functional and phenotypic correction of blood cells: measured by chromosomal stability of T-lymphocytes in the presence of diepoxybutane (DEB)
Hematologic correction: measured by changes in previously declining pre-treatment blood count trajectories
Other measured parameters include safety, vector integration profile, and clonal repertoire.

Updated Results Presented at ASGCT (Free ASGCT Whitepaper) 2018:

Data presented today includes all five patients treated to date with RP-L102 under academic protocol:

All patients demonstrated continued improvement in engraftment following administration of RP-L102. In patient 02002—the first patient treated with higher doses—peripheral blood VCN increased to 44% at 24 months, from 34% at 19 months and 17% at 12 months.
Sustained phenotypic reversals and earlier evidence of gene correction were seen in higher-dosed patients (02002 and 02006) within months of treatment. Notably, based on MMC and DEB assays, these two patients showed durable improvements consistent with somatic mosaicism that has persisted over the course of several months. Somatic mosaicism is an FA phenomenon where patients largely do not develop the typical FA manifestations of bone marrow failure and leukemia1. Moreover, in patient 02002, the bone marrow resistance to MMC increased to 70% at 24 months (up from ~20% at 12 months), approaching the phenotype of a healthy donor.
Patients 02004 and 02005 received non-optimized and lower doses of RP-L102. Nonetheless, evidence of gene-corrected and phenotypically-normalized cells was seen, but after longer durations.
One patient (01003) received RP-L102 manufactured in the presence of transduction enhancers. Based on early data, RP-L102 transduction efficiency (drug product VCN) was the highest to date—more than five-fold higher compared to the best previously achieved (0.53 for patient 2006 and 0.43 for patient 2002). Additionally, early engraftment accelerated more than three-fold compared to earlier patients.
No serious drug-related adverse events have been observed to date.
"We are very pleased by the trajectory of progressively increasing gene markings and reversion to a phenotype where the blood and bone marrow cells are resistant to DNA damaging agents. Moreover, it appears that the stabilization of blood counts, which previously were declining, resulted from an increase in gene-corrected peripheral blood cell lineages," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "Moving forward, we plan to use a further optimized process with the addition of transduction enhancers and treat patients earlier in their disease course. These modifications are expected to enable more robust responses."

"The value creation we seek, relative to standard transplant, is to enable intervention soon after diagnosis as a preventative measure. Therefore, stability of blood counts is going to be an important indicator of the potential benefit of our FANCA-focused LVV gene therapy programs for this life-threatening disease," continued Dr. Shah. "Rocket remains committed to advancing the standard of care in FA, and to the continued advancement of our pipeline of five LVV and AAV-based gene therapy programs. We will continue to innovate and aspire to create new options for patients with devastating diseases."

Additional patient data from the FA program is expected over the next 12-18 months. Based on these promising preliminary results, Rocket will engage with regulatory authorities to progress RP-L102 towards a potential global registrational study in 2019.

Presentations from ASGCT (Free ASGCT Whitepaper) will be posted on Rocket’s website at: www.rocketpharma.com/pipeline/. Updated data from the ongoing Phase 1/2 trial in FA will be submitted for publication.

About RP-L102 (LVV-based gene therapy for Fanconi Anemia):

RP-L102 is Rocket’s lentiviral vector (LVV)-based gene therapy in development for patients with FA with Rocket’s collaboration partners at Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain, CIBER-Rare Diseases and IIS-Fundación Jiménez Díaz. The International Fanconi Anemia Gene Therapy Working Group helped develop the structure of RP-L102, which begins with a HIV-1-derived, self-inactivating lentiviral vector. RP-L102’s lentiviral vector carries the FANC-A gene as part of the PGK-FANCA-WPRE expression cassette which includes a phosphoglycerate kinase (PKG) promoter and an optimized woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The ex vivo administration process begins with the removal and isolation of hematopoietic stem cells using a CD34+ selection process. Autologous genetically modified CD34+ enriched hematopoietic cells (fresh or cryopreserved) are infused back into patients to restore function. RP-L102 is currently being studied in a Phase 1/2 clinical trial in the European Union with an Investigational Medicinal Product Dossier (IMPD) in place with the Spanish Agency for Medicines and Health Products. RP-L102 has been granted Orphan Drug designation for the treatment of Fanconi Anemia type A in the United States and in Europe.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning, which is highly toxic for the patient. HSCT is frequently complicated by graft versus host disease and also increases the risk of many solid organ malignancies. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the ‘gold standard’ test for FA diagnosis. The DEB assay can further differentiate FA patients from somatic mosaic patients. Somatic mosaicism occurs when there is a spontaneous reversion mutation that can lead to a mixed chimerism of corrected and uncorrected bone marrow cells leading to stabilization or correction of an FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism provides strong rationale for the development of FA gene therapy and demonstrates the selective advantage of gene-corrected hematopoietic cells in FA1.

Stemline Therapeutics Announces Three SL-401 Clinical Presentations, Including an Oral Presentation, at the Upcoming EHA Congress

On May 18, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that SL-401 will be the subject of three clinical presentations, including an oral presentation on the pivotal BPDCN program (Press release, Stemline Therapeutics, MAY 18, 2018, View Source [SID1234526804]). Updated data from the ongoing Phase 2 trial in chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF) will also be presented. Presentations will be delivered at the upcoming 23rdCongress of the European Hematology Association (EHA) (Free EHA Whitepaper), to be held from June 14-17, 2018, in Stockholm, Sweden. Abstracts are now available on the EHA (Free EHA Whitepaper) congress website.

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!

Details on the presentations are as follows:

Results of Pivotal Phase 2 Trial of SL-401 in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm
• Abstract: S116
• Session: Miscellaneous Treatments in AML
• Presenter: Naveen Pemmaraju, MD; MD Anderson Cancer Center
• Oral Presentation: Friday, June 15; 11:45 – 12:00 CEST (5:45 AM – 6:00 AM ET)
• Location: Room A4

Results from Ongoing Phase 1/2 Trial of SL-401 in Patients with Intermediate or High Risk Relapsed/Refractory Myelofibrosis
• Abstract: PF618
• Session: Myeloproliferative neoplasms – Clinical
• Poster Presentation: Friday, June 15; 17:30 – 19:00 CEST (11:30 AM – 1 PM ET)
• Location: Poster Area

Results from Ongoing Phase 1/2 Trial of SL-401 in Patients with Relapsed/Refractory CMML
• Abstract: PF626
• Session: Myeloproliferative neoplasms – Clinical
• Poster Presentation: Friday, June 15; 17:30 – 19:00 CEST (11:30 AM – 1 PM ET)
• Location: Poster Area
Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, commented, "We are honored to be showcasing, via oral presentation, the results of our SL-401 pivotal trial in BPDCN to a European audience via the EHA (Free EHA Whitepaper) Congress. We believe this selection underscores SL-401’s robust clinical data and increased global awareness of BPDCN, a devastating malignancy of high unmet medical need. In the U.S., we remain on track to complete our rolling Biologics License Application (BLA) submission this quarter. In Europe, we anticipate feedback later this year from the European Medicines Agency (EMA) regarding the timing of a potential regulatory filing in the European Union. In addition, we and our investigators continue to report encouraging signs of clinical activity and safety in indications beyond BPDCN, including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), and we look forward to presenting updated data from these indications at the conference."

Following each presentation at the conference, the data presented will be available on Stemline’s website (www.stemline.com) under the Scientific Presentations tab.

About BPDCN
Please visit the BPDCN disease awareness booth (#4125) at ASCO (Free ASCO Whitepaper) 2018 and www.bpdcninfo.com.

Janssen to Present New Data in Urothelial, Haematologic and Prostate Cancers at ASCO 2018, including Best of ASCO Selections

On May 18, 2018 The Janssen Pharmaceutical Companies of Johnson & Johnson, reported 21 company-sponsored abstracts will be presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL on June 1-5 (Press release, Johnson & Johnson, MAY 18, 2018, View Source [SID1234526805]). New data analyses in support of a portfolio of products, including the investigational treatments erdafitinib and apalutamide, as well as approved treatments Imbruvica (ibrutinib), Darzalex (daratumumab), and Zytiga (abiraterone acetate) will be highlighted across urothelial, haematologic and prostate 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!

Notably, Phase 2 trial results for the investigational compound erdafitinib, which received U.S. Food and Drug Administration (FDA) Breakthrough Therapy Designation, will be presented during an oral presentation on Sunday, June 3 (Abstract #4503).1,2 For haematologic cancers, Phase 3 data from the iNNOVATE study will provide the first look at ibrutinib plus rituximab versus placebo plus rituximab in patients with newly diagnosed and relapsed/refractory Waldenström’s macroglobulinemia (WM) (Abstract #8003).3 In addition, Phase 2 data from the CAPTIVATE study will be presented evaluating ibrutinib plus venetoclax in first-line chronic lymphocytic leukaemia (CLL) (Abstract #7502).4 Oral presentations for erdafitinib and ibrutinib have been selected to be featured at the Best of ASCO (Free ASCO Whitepaper) 2018 Meetings, which highlight cutting-edge science and reflect the leading research in oncology.

"The breadth of new data from our portfolio shows our commitment to finding solutions for patients living with cancer according to their specific treatment needs," said Dr Ivo Winiger-Candolfi, Oncology Therapeutic Area Lead, Janssen Europe, Middle East and Africa. "It reinforces our dedication to work with our partners and move a step closer to making cancer a preventable, chronic or curable disease."

Selected data presentations include:

Erdafitinib: Results from the primary analysis of the Phase 2 study of erdafitinib (ERDA; JNJ-42756493) in patients with metastatic or unresectable urothelial carcinoma (mUC) and Fibroblast Growth Factor Receptor alterations (FGFRalt).
These data will be featured in an oral presentation from 9:00 – 9:12 a.m. CDT on Sunday, June 3 (Abstract #4503)1 and have been selected for the Best of ASCO (Free ASCO Whitepaper) 2018 Meetings.
Ibrutinib: Findings from the Phase 3 placebo-controlled iNNOVATE study will be presented, assessing ibrutinib plus rituximab versus placebo plus rituximab in patients with newly diagnosed and relapsed/refractory WM.*
These data will be featured in an oral presentation from 3:45 – 3:57 p.m. CDT on Friday, June 1 (Abstract #8003)3 and have been selected for the Best of ASCO (Free ASCO Whitepaper) 2018 Meetings.
Ibrutinib: Early results from the Phase 2 CAPTIVATE study will be presented, evaluating ibrutinib in combination with venetoclax in first-line CLL.*
These data will be featured in an oral presentation from 10:09 – 10:21 a.m. CDT on Sunday, June 3 (Abstract #7502) and have been selected for the Best of ASCO (Free ASCO Whitepaper) 2018 Meetings.4
Daratumumab: Phase 1 data from the MMY1001 study will report on the efficacy and safety of daratumumab in combination with carfilzomib and dexamethasone in lenalidomide-refractory patients with relapsed multiple myeloma.
These data will be presented in an oral presentation from 3:09 – 3:21 p.m. CDT on Friday, June 1 (Abstract #8002).5
Daratumumab: Follow-up efficacy and safety data from the pivotal Phase 3 ALCYONE study will be presented for daratumumab in combination with bortezomib, melphalan and prednisone in patients with newly diagnosed multiple myeloma who are transplant ineligible.
These data will be presented in a poster presentation from 8:00 – 11:30 a.m. CDT on Monday, June 4 (Abstract #8031).6
Daratumumab: Safety run-in results from the Phase 3 ANDROMEDA study will be presented evaluating the subcutaneous use of daratumumab in combination with cyclophosphamide, bortezomib, and dexamethasone in patients with newly diagnosed amyloid light chain (AL) amyloidosis.7 Amyloidosis is an incurable disease in which cells that normally produce antibodies make an abnormal protein that deposits in and causes damage to organs such as the heart and kidneys.8
These data will be presented in a poster discussion presentation from 3:00 – 4:15 p.m. CDT on Monday, June 4 (Abstract #8011).
Apalutamide: New analyses from the pivotal Phase 3 SPARTAN clinical trial will be presented examining the relationship between time to metastasis (TTM) and site of metastases in patients with non-metastatic castration-resistant prostate cancer (nmCRPC).
These data will be presented in a poster presentation from 1:15 – 4:45 p.m. CDT on Saturday, June 2 (Abstract #5033).9
Abiraterone acetate: New findings from the pivotal Phase 3 LATITUDE clinical trial in patients with metastatic high-risk castration-sensitive prostate cancer (CSPC) will be presented.
These data will be presented in a poster presentation from 1:15 – 4:45 p.m. CDT on Saturday, June 2 (Abstract #5028).10
Prostate Cancer: New analysis exploring the association between metastasis-free survival (MFS) and overall survival (OS) will be presented in nmCRPC for the first time.
These data will be presented in a poster presentation from 1:15 – 4:45 p.m. CDT on Saturday, June 2 (Abstract #5032).11
For more information on the abstracts presented by Janssen, please click here.

*Abstracts were submitted by ibrutinib co-developer partner, Pharmacyclics, an AbbVie company.

#ENDS#

About erdafitinib

Erdafitinib is an investigational, once-daily pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor being evaluated by Janssen Research and Development in Phase 2 and 3 clinical trials in patients with advanced urothelial cancer and other solid tumours. FGFRs are a family of receptor tyrosine kinases which may be upregulated in various tumour cell types and may be involved in tumour cell differentiation and proliferation, tumour angiogenesis, and tumour cell survival.12 In 2008, Janssen entered into an exclusive worldwide license and collaboration agreement with Astex Therapeutics Ltd. to develop and commercialise erdafitinib.

About ibrutinib

Ibrutinib is a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor, which works by forming a strong covalent bond with BTK to block the transmission of cell survival signals within the malignant B-cells.13 By blocking this BTK protein, ibrutinib helps kill and reduce the number of cancer cells, thereby delaying progression of the cancer.14

Ibrutinib is currently approved in Europe for the following uses:15

Chronic lymphocytic leukaemia (CLL): As a single agent for the treatment of adult patients with previously untreated CLL, and as a single agent or in combination with bendamustine and rituximab (BR) for the treatment of adult patients with CLL who have received at least one prior therapy.
Mantle cell lymphoma (MCL): Adult patients with relapsed or refractory mantle cell MCL.
Waldenström’s macroglobulinemia (WM): Adult patients who have received at least one prior therapy or in first-line treatment for patients unsuitable for chemo-immunotherapy.
The most common adverse reactions seen with ibrutinib include diarrhoea, neutropenia, haemorrhage (e.g., bruising), musculoskeletal pain, nausea, rash, and pyrexia.15

For a full list of side effects and for further information on dosage and administration, contraindications and other precautions when using ibrutinib please refer to the Summary of Product Characteristics for further information.15

About daratumumab

Daratumumab is a first-in-class biologic targeting CD38, a surface protein that is highly expressed across multiple myeloma cells, regardless of disease stage.16,17,18 Daratumumab is believed to induce tumour cell death through multiple immune-mediated mechanisms of action, including complement-dependent cytotoxicity (CDC), antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), as well as through apoptosis, in which a series of molecular steps in a cell lead to its death.19 A subset of myeloid derived suppressor cells (MDSCs), CD38+ regulatory T cells (Tregs) and CD38+ B cells (Bregs) were decreased by daratumumab.19 Daratumumab is being evaluated in a comprehensive clinical development program across a range of treatment settings in multiple myeloma, such as in frontline and relapsed settings.20,21,22,23,24,25,26,27,28 Additional studies are ongoing or planned to assess its potential for a solid tumour indication and in other malignant and pre-malignant diseases in which CD38 is expressed, such as smouldering myeloma.29,30,31,32 For more information, please see www.clinicaltrials.gov.

Daratumumab is currently approved in Europe for the following uses:19

As monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a proteasome inhibitor and an immunomodulatory agent and who have demonstrated disease progression on the last therapy.
In combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.
The most common adverse reactions seen with daratumumab include infusion reactions, fatigue, nausea, diarrhoea, muscle spasms, pyrexia, cough, dyspnoea, neutropenia, thrombocytopenia and upper respiratory tract infection. In addition, in combination with bortezomib, peripheral oedema and peripheral sensory neuropathy were frequently reported.19

For a full list of side effects and for further information on dosage and administration, contraindications and other precautions when using daratumumab please refer to the Summary of Product Characteristics.19

In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialise daratumumab.33

About apalutamide

Apalutamide is an investigational, next-generation oral androgen receptor (AR) inhibitor that blocks the androgen signalling pathway in prostate cancer cells.34 Apalutamide inhibits the growth of cancer cells in three ways: by preventing the binding of androgen to the AR; by stopping the AR from entering the cancer cells; and by preventing the AR from binding to the DNA of the cancer cell.34 Apalutamide received US FDA approval on February 14, 2018 for the treatment of non-metastatic CRPC.35 On February 9, 2018 Janssen submitted a Marketing Authorisation Application to the European Medicines Agency (EMA).36

About abiraterone acetate

Abiraterone acetate plus prednisone / prednisolone is the only approved therapy in mCRPC that inhibits production of androgens (which fuel prostate cancer growth) at all three sources that are important in prostate cancer – the testes, adrenals and the tumour itself.37,38

Abiraterone acetate with prednisone / prednisolone is currently approved in Europe for the following uses:37

The treatment of newly diagnosed high risk metastatic hormone sensitive prostate cancer (mHSPC) in adult men in combination with androgen deprivation therapy (ADT).
The treatment of metastatic castration resistant prostate cancer (mCRPC) in adult men who are asymptomatic or mildly symptomatic after failure of androgen deprivation therapy in whom chemotherapy is not yet clinically indicated.
The treatment of mCRPC in adult men whose disease has progressed on or after a docetaxel-based chemotherapy regimen.
The most common adverse reactions seen with abiraterone acetate plus prednisone / prednisolone include urinary tract infection, hypokalemia, hypertension, and peripheral oedema.37

For a full list of side effects and for further information on dosage and administration, contraindications and other precautions when using abiraterone acetate plus prednisone / prednisolone please refer to the Summary of Product Characteristics.37