On December 5, 2019 FerGene, a new gene therapy company formed by Ferring Pharmaceuticals and Blackstone Life Sciences, reported positive results from the pivotal Phase 3 clinical trial evaluating nadofaragene firadenovec (rAd-IFN/Syn3), an investigational gene therapy, for the treatment of high-grade, Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC) (Press release, FerGene, DEC 5, 2019, View Source [SID1234551976]). FKD Therapies Oy (FKD) has led the development and regulatory filing of nadofaragene firadenovec, which has been studied in 33 centers across the U.S. in collaboration with the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC). The results were presented during the bladder cancer session at the Society of Urologic Oncology 20th Annual Meeting in Washington D.C.

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"These data were part of our submission package to the FDA, and we look forward to continuing to work with the agency to potentially bring nadofaragene firadenovec to patients with BCG unresponsive disease."

The Phase 3 study of 157 patients from the U.S. met its primary endpoint with 53% of CIS ± Ta/T1 patients (carcinoma in situ; bladder cancer that is confined to the superficial layer, with or without concomitant high-grade Ta or T1 papillary disease) achieving a CR at three months, and 24% continuing to show a CR at 12 months. Moreover, the study also demonstrated broad efficacy in this difficult to treat patient population with a 73% HGRF survival in patients with papillary disease at three months and 44% HGRF survival at 12 months. In the study, nadofaragene firadenovec was instilled directly into the patients’ bladder every three months. All responses at 12 months were confirmed by protocol-mandatory five-point biopsies.

Bladder cancer is one of the most frequently occurring cancers with an estimated 699,450 people living with bladder cancer and more than 80,000 new cases diagnosed each year in the U.S. alone.1 In high-grade NMIBC patients, BCG is the standard treatment, and, although effective, over 60% of these tumors eventually re-occur. 2,3

"Currently, patients living with high-grade NMIBC who are unresponsive to BCG have few treatment options and often face bleak outcomes, including complete bladder removal, known as cystectomy," said Colin P. N. Dinney, MD, Professor and Chairman of the Department of Urology at The University of Texas M.D. Anderson Cancer Center. "Cystectomy is a complex and life-altering surgical procedure for patients, so these positive results from the Phase 3 trial of nadofaragene firadenovec are highly promising for patients. It would be gratifying to provide an alternative that addresses the critical unmet need for effective second-line therapy for patients facing radical cystectomy."

In the Phase 3 trial, the most common adverse events (AEs) included fatigue, bladder spasm and discharge around the catheter, micturition urgency, hematuria, chills, fever, headache, painful urination, urinary tract infection, and diarrhea. No grade 4 or 5 treatment-related AEs were reported in the study. Study drug-related AEs were transient and local in nature, with a median duration of less than two days, with the exception of fatigue, which had a median duration of 11 days and urinary frequency which had a median duration of 41 days. There was a 1.9% percent rate of discontinuations due to study drug-related AEs.

"We are pleased with these Phase 3 data results, including the complete response rates and favorable safety profile seen with nadofaragene firadenovec," said Nigel R. Parker4, PhD, of FKD Therapies Oy. "These data were part of our submission package to the FDA, and we look forward to continuing to work with the agency to potentially bring nadofaragene firadenovec to patients with BCG unresponsive disease."

"As a practicing urologist and trial investigator, it’s encouraging to see these types of efficacy and safety results in patients with high-grade NMIBC, an area that’s been in need of new innovative treatment options for more than 20 years," said Neal Shore, MD, FACS, Medical Director, Carolina Urologic Research Center. "These robust clinical results further demonstrate the potential of nadofaragene firadenovec as a valuable treatment option for NMIBC patients."

The U.S. Food and Drug Administration (FDA) has validated FKD’s Biologics License Application (BLA) and granted Priority Review for nadofaragene firadenovec, which previously received Fast Track and Breakthrough Therapy Designations.

About nadofaragene firadenovec

Nadofaragene firadenovec (rAd-IFN/Syn3) is an investigational gene therapy being developed as a treatment for patients with high-grade, BCG unresponsive, NMIBC. It is an adenovirus vector-based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder every three months. The vector enters the cells of the bladder wall, where, it breaks down, releasing the active gene to do its work. The internal gene/DNA machinery of the cells ‘picks up’ the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patient’s own bladder wall cells into multiple interferon microfactories, enhancing the body’s natural defenses against the cancer.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is an early form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.5 It is estimated that there will be 80,000 new cases of bladder cancer in the U.S. in 2019; more than 70% of these cases present as NMIBC.2,6 In patients with high-grade NMIBC, intravesical BCG is the recommended treatment; however, between 30% and 50% cases with high-grade disease will recur.7 The outcome for BCG unresponsive patients is poor, with total cystectomy (complete removal of the bladder) often being the next treatment option.8

On December 5, 2019 NeuClone Pharmaceuticals Ltd (NeuClone), a clinical-stage biopharmaceutical company exclusively focused on developing high-quality biosimilar products, reported that NeuCeptin, a biosimilar candidate of Herceptin (trastuzumab), has successfully met all primary and secondary endpoints in a Phase I clinical trial (Press release, NeuClone Pharmaceuticals, DEC 5, 2019, View Source [SID1234551975]). This includes all pre-specified criteria demonstrating clinical pharmacokinetic (PK) similarity of NeuCeptin, compared to US- and EU-sourced Herceptin. Additionally, the safety and tolerability profiles were equivalent between all three treatment arms.

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Successful completion of the Phase I trial is a significant milestone in the development of a biosimilar as Phase II trials and in some instances Phase III trials are not required to achieve regulatory approval. As of March 2019, no biosimilar found to be highly similar in analytical and human PK studies failed to be approved in the US, EU, Canada or Australia due to clinical inequivalence.1

NeuCeptin is one of several biosimilars developed in partnership between NeuClone and Serum Institute of India Pvt Ltd (Serum Institute), and references trastuzumab, a HER2-targeting monoclonal antibody approved by the FDA and EMA to treat HER2-positive breast and gastric cancer. Trastuzumab was originally commercialised under the brand name Herceptin and generated global sales of USD 7.0 billion in 2018.2

"Positive results from the NeuCeptin trial reflect our dedication to provide high-quality, affordable biologics to a greater number of patients," stated Dr Noelle Sunstrom, CEO and Founder of NeuClone. "Pharmacokinetics are pivotal to the demonstration of biosimilarity and these successful results greatly de-risk this particular program and also validate NeuClone and Serum Institute’s development approach to be replicated for many biosimilars in our pipeline."

"It is extremely pleasing to see the progress of our biosimilar portfolio with co-development partner, NeuClone. We have now achieved successful Phase I results of NeuCeptin (trastuzumab), nearly completed dosing of the Phase I trial for NeuLara (ustekinumab), and palivizumab and pertuzumab biosimilars are both progressing into preclinical and clinical testing," stated Mr. Adar Poonawalla, CEO of Serum Institute. "This clearly speaks of our commitment to make highly cost-effective biosimilars available to all."

NeuClone has 20 biosimilars in various stages of development including clinical-stage candidates NeuCeptin (trastuzumab) and NeuLara (ustekinumab). Several other biosimilars are set to begin clinical development over the coming years. As part of its strategy for biosimilar products, NeuClone remains open to potential development and commercialisation collaborations.

About the Study
The Phase I clinical trial (registration number ACTRN12618001657213) was a randomised, double-blind, single-dose, three-arm study to evaluate the pharmacokinetics (PK) and safety of NeuCeptin compared to Herceptin (trastuzumab). Over 100 healthy volunteers were enrolled in sites across Australia and randomised (1:1:1) to receive either NeuCeptin biosimilar, Herceptin sourced from the US, or Herceptin sourced from the EU, administered as a single intravenous infusion. PK endpoints included: area under the concentration-time curve from first to last timepoint measured (AUC0-last), area under the concentration-time curve from time zero extrapolated to infinity (AUC0-inf), and maximum serum concentration (Cmax). The 90% confidence intervals of each endpoint were contained within pre-specified bioequivalence margins of 80% to 125%, for all 3 pairwise comparisons. The trial was conducted under the Therapeutic Goods Administration (TGA) Clinical Trial Notification (CTN) scheme offering a streamlined approach with data output supported by global regulatory agencies such as the EMA and US FDA.

On December 5, 2019 Akoya Biosciences, Inc., The Spatial Biology Company, reported the completion of a $50 million financing from both new and existing investors (Press release, Akoya Biosciences, DEC 5, 2019, View Source [SID1234551974]). The round will fund significant growth through expansion of commercial and operational resources as well as continued product development of Akoya’s CODEX and Phenoptics platforms for spatial biology for research and clinical markets.

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The financing was led by Piper Jaffray Merchant Banking, a division of Piper Jaffray, with continued participation from Telegraph Hill Partners. New strategic partners also include Agilent Technologies and Innovatus Capital Partners, LLC. This financing follows Akoya’s September 2018 Series C funding used to support the acquisition of the Phenoptics Portfolio from PerkinElmer.

Brian McKelligon, Chief Executive Officer of Akoya, will present on Thursday, December 5, 2019 at the Piper Jaffray 31st Annual Healthcare Conference taking place at the Lotte New York Palace, New York. The session will be held in the SoHo Track – Hubbard 1, 5th floor from 2:10-2:30 PM.

"2019 was an exciting year of growth for Akoya, led by new product introductions for both CODEX and Phenoptics platforms," said Mr. McKelligon. "This support from industry leading investors and partners provides the vital endorsement and resources necessary for Akoya to continue to deliver innovative tools to enable a better understanding of the pathophysiology of cancer and provide more informative results for clinical decisions.

Thomas Schnettler, managing director at Piper Jaffray, has joined Akoya’s Board of Directors and commented, "Akoya is transforming how immunotherapies are developed, from discovery of novel biomarkers in the tumor microenvironment, to deploying high throughput, highly sensitive information to direct patient therapies." Schnettler continued, "Our investment represents our confidence in Akoya’s relentless execution to deliver the next generation of pathology tools."

"We are excited to support Akoya’s efforts to commercialize multispectral imaging solutions to unlock the full potential of spatial biology," said Claes Ekstrom, Managing Director at Innovatus.

The new financing will enable further product development of the CODEX and Phenoptics platforms for multiplexed immunofluorescence imaging and analysis, commercial expansion, and the scale up of operations and manufacturing. These expansions are driven by dramatic growth in the immuno-oncology market and mounting evidence that spatial analysis using multiplex immunofluorescence is critical to understanding cancer’s complexity, and potentially provides more power for predicting patient response to immunotherapies.

On December 5, 2019 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB), a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that it will host an R&D event and live webcast featuring several key opinion leaders ("KOLs") to discuss the Company’s advanced antibody-based therapeutic pipeline and recent clinical and corporate developments on Wednesday, December 11, 2019 from 12:00-2:00pm EST in New York City (Press release, Y-mAbs Therapeutics, DEC 5, 2019, View Source [SID1234551972]).

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The event will feature presentations by KOLs Dr. Shakeel Modak, M.D., MRCP, Memorial Sloan Kettering Cancer Center ("MSK"), Dr. Kim Kramer, M.D., MSK, and Dr. Jaume Mora, M.D., Ph.D., SJD Barcelona Children’s Hospital, who will discuss the current treatment landscape and unmet medical needs for treating patients with high-risk pediatric neuroblastoma and other solid tumors. Drs. Modak, Kramer, and Mora will be available to answer questions at the conclusion of the event.

Y-mAbs management will also provide an in-depth overview of the Company’s broad and advanced product pipeline as well as a review of recent corporate and clinical developments. Y-mAbs is advancing two pivotal-stage product candidates, naxitamab and omburtamab, targeting tumors that express GD2 and B7-H3, respectively.

Members of the media and public may access the event via a live webcast.

On December 5, 2019 OncoSec Medical Incorporated ("OncoSec") (Nasdaq: ONCS), a company developing late-stage intratumoral cancer immunotherapies, reported it will present interim efficacy, immunological and safety data from its ongoing KEYNOTE-890 study of TAVO in combination with KEYTRUDA in patients with late-stage, heavily pretreated, chemo-refractory metastatic triple negative breast cancer (mTNBC) at the upcoming San Antonio Breast Cancer Symposium (SABCS) Annual Meeting being held December 10-14, 2019 (Press release, OncoSec Medical, DEC 5, 2019, View Source [SID1234551971]).

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The poster, titled "Phase 2, open-label study of intratumoral tavokinogene telseplasmid (tavo) plus electroporation in combination with intravenous pembrolizumab therapy in patients with inoperable locally advanced or metastatic triple negative breast cancer (mTNBC) (KEYNOTE- 890/OMS-I141)," will be presented by lead author Melinda L. Telli, MD on Thursday, December 12th from 5:00 p.m. – 7:00 p.m. CST.

The SABCS abstracts are listed on the conference website under Abstracts at View Source!/7946 .