ESSA Pharma Announces Multiple Abstracts Accepted for Presentation at Upcoming Medical and Scientific Symposia

On February 10, 2020 ESSA Pharma Inc. (Nasdaq: EPIX; TSX-V: EPI), a pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, reported that several abstracts on ESSA’s lead clinical candidate, EPI-7386, have been selected for presentation at upcoming medical and scientific symposia (Press release, ESSA, FEB 10, 2020, View Source [SID1234554112]). These presentations will provide further preclinical characterization of EPI-7386 including new preclinical data regarding safety studies, gene expression and combination data with antiandrogens.

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2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium

Title: IND Candidate EPI-7386 as an N-terminal Domain Androgen Receptor Inhibitor in Development for the Treatment of Prostate Cancer

Abstract #:

142

Presenter:

Dr. Ronan Le Moigne

Session Title:

Prostate Cancer

Poster Board:

F22

Date:

Thursday, February 13, 2020

Time:

11:30am – 1:00pm PST

Location:

Moscone West Building, San Francisco, CA

American Association for Cancer Research Special Conference on Advances in Prostate Cancer Research

Title: The N-terminal Domain Inhibitor of the Androgen Receptor, EPI-7386, Targets Full Length and Splice Variant Driven Pathways

Presenter:

Dr. Nan Hyung Hong

Poster Session:

B

Date:

Saturday, March 14, 2020

Time:

12:30pm – 3:00pm MST

Location:

Grand Hyatt Denver, Denver, CO

2020 American Urological Association Annual Meeting

Title: The Preclinical Characterization and Development of EPI-7386, an N-terminal domain androgen receptor inhibitor, for the treatment of prostate cancer

Abstract #:

20-6346

Presenter:

Dr. Ronan Le Moigne

Session Title:

Prostate Cancer: Advanced IV

Moderated Poster:

MP79

Date:

Monday, May 18, 2020

Time:

9:30am – 11:30am EST

Location:

Walter E. Washington Convention Center, Washington, D.C.

Eisai to Present Latest on LENVIMA® (lenvatinib) at the 2020 Genitourinary Cancers Symposium

On February 10, 2020 Eisai reported the presentation of three abstracts at the 2020 Genitourinary Cancers Symposium (#GU20) in San Francisco from February 13-15 (Press release, Eisai, FEB 10, 2020, View Source [SID1234554111]). New data to be presented on lenvatinib (marketed as LENVIMA) include results from a Phase 2 trial evaluating the efficacy and tolerability of lenvatinib plus everolimus (LEN+EVE) in patients with non-clear cell renal cell carcinoma (nccRCC) who had not previously received chemotherapy for advanced disease, and an analysis of genomic biomarkers via whole exome sequencing and outcomes in patients with advanced renal cell carcinoma (RCC) who were treated with pembrolizumab plus lenvatinib in Study 111/KEYNOTE-146.

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A trials in progress poster on the LEAP-011 phase 3 study assessing the efficacy and safety of pembrolizumab plus lenvatinib, compared with pembrolizumab plus placebo in patients with advanced urothelial carcinoma (UC) will also be shared.

"We look forward to sharing new investigational information that expand our understanding of LENVIMA in advanced renal cell carcinoma," said Dr. Takashi Owa, Vice President, Chief Medicine Creation Officer and Chief Discovery Officer, Oncology Business Group at Eisai. "We are committed to deepening our understanding of our medicines and researching combinations for our patients."

This release discusses investigational uses for FDA-approved products. It is not intended to convey conclusions about efficacy and safety. There is no guarantee that any investigational uses of FDA-approved products will successfully complete clinical development or gain FDA approval.

The full list of Eisai presentations along with the time and location of each session is included below:

Abstract Name

Session (All times are U.S. Pacific Standard Time)

Phase III study of first-line pembrolizumab
(pembro) plus lenvatinib (len) in patients (pts) with
advanced urothelial carcinoma (UC) ineligible for
platinum-based chemotherapy: LEAP-011.

Poster Session B

Abstract # TPS597 / Poster Board #N15

Friday, February 14, 2020

12:15-1:45 PM and 5:15-6:15 PM

Yohann Loriot, MD, PhD

A phase II study of lenvatinib plus everolimus in
patients with advanced non-clear cell renal cell
carcinoma (nccRCC): Study 221.

Poster Session C

Abstract # 685 / Poster Board G5

Saturday, February 15, 2020

7:00 AM-7:55 AM and 12:30 PM-2:00 PM

Thomas Hutson, DO, FACP, PharmD

Genomic biomarkers of response to
lenvatinib/pembrolizumab (Len/Pembro) in patients
with advanced renal cell carcinoma: Study 111.

Poster Session C

Abstract # 733 / Poster Board J9

Saturday, February 15, 2020

7:00 AM- 7:55 AM and 12:30 PM-2:00 PM

Chung-Han Lee, MD, PhD

In March 2018, Eisai and Merck (known as MSD outside the United States and Canada), through an affiliate, entered into a strategic collaboration for the worldwide co-development and co-commercialization of lenvatinib, both as monotherapy and in combination with Merck’s anti-PD-1 therapy pembrolizumab.

About LENVIMA (lenvatinib) Capsules 10 mg and 4 mg

LENVIMA is indicated:

For the treatment of patients with locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer (RAI-refractory DTC)
In combination with everolimus, for the treatment of patients with advanced renal cell carcinoma (RCC) following one prior anti-angiogenic therapy
For the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
Important Safety Information

Warnings and Precautions

Hypertension. In DTC, hypertension occurred in 73% of patients on LENVIMA (44% grade 3-4). In RCC, hypertension occurred in 42% of patients on LENVIMA + everolimus (13% grade 3). Systolic blood pressure ≥160 mmHg occurred in 29% of patients, and 21% had diastolic blood pressure ≥100 mmHg. In HCC, hypertension occurred in 45% of LENVIMA-treated patients (24% grade 3). Grade 4 hypertension was not reported in HCC.

Serious complications of poorly controlled hypertension have been reported. Control blood pressure prior to initiation. Monitor blood pressure after 1 week, then every 2 weeks for the first 2 months, and then at least monthly thereafter during treatment. Withhold and resume at reduced dose when hypertension is controlled or permanently discontinue based on severity.

Cardiac Dysfunction. Serious and fatal cardiac dysfunction can occur with LENVIMA. Across clinical trials in 799 patients with DTC, RCC, and HCC, grade 3 or higher cardiac dysfunction occurred in 3% of LENVIMA treated patients. Monitor for clinical symptoms or signs of cardiac dysfunction. Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity

Arterial Thromboembolic Events. Among patients receiving LENVIMA or LENVIMA + everolimus, arterial thromboembolic events of any severity occurred in 2% of patients in RCC and HCC and 5% in DTC. Grade 3-5 arterial thromboembolic events ranged from 2% to 3% across all clinical trials. Permanently discontinue following an arterial thrombotic event. The safety of resuming after an arterial thromboembolic event has not been established and LENVIMA has not been studied in patients who have had an arterial thromboembolic event within the previous 6 months.

Hepatotoxicity. Across clinical studies enrolling 1,327 LENVIMA-treated patients with malignancies other than HCC, serious hepatic adverse reactions occurred in 1.4% of patients. Fatal events, including hepatic failure, acute hepatitis and hepatorenal syndrome, occurred in 0.5% of patients. In HCC, hepatic encephalopathy occurred in 8% of LENVIMA-treated patients (5% grade 3-5). Grade 3-5 hepatic failure occurred in 3% of LENVIMA-treated patients. 2% of patients discontinued LENVIMA due to hepatic encephalopathy and 1% discontinued due to hepatic failure.

Monitor liver function prior to initiation, then every 2 weeks for the first 2 months, and at least monthly thereafter during treatment. Monitor patients with HCC closely for signs of hepatic failure, including hepatic encephalopathy. Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity.

Renal Failure or Impairment. Serious including fatal renal failure or impairment can occur with LENVIMA. Renal impairment was reported in 14% and 7% of LENVIMA-treated patients in DTC and HCC, respectively. Grade 3-5 renal failure or impairment occurred in 3% of patients with DTC and 2% of patients with HCC, including 1 fatal event in each study. In RCC, renal impairment or renal failure was reported in 18% of LENVIMA + everolimus–treated patients (10% grade 3).

Initiate prompt management of diarrhea or dehydration/hypovolemia. Withhold and resume at reduced dose upon recovery or permanently discontinue for renal failure or impairment based on severity.

Proteinuria. In DTC and HCC, proteinuria was reported in 34% and 26% of LENVIMA-treated patients, respectively. Grade 3 proteinuria occurred in 11% and 6% in DTC and HCC, respectively. In RCC, proteinuria occurred in 31% of patients receiving LENVIMA + everolimus (8% grade 3). Monitor for proteinuria prior to initiation and periodically during treatment. If urine dipstick proteinuria ≥2+ is detected, obtain a 24-hour urine protein. Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity.

Diarrhea. Of the 737 LENVIMA-treated patients in DTC and HCC, diarrhea occurred in 49% (6% grade 3). In RCC, diarrhea occurred in 81% of LENVIMA + everolimus–treated patients (19% grade 3). Diarrhea was the most frequent cause of dose interruption/reduction, and diarrhea recurred despite dose reduction. Promptly initiate management of diarrhea. Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity.

Fistula Formation and Gastrointestinal Perforation. Of the 799 patients treated with LENVIMA or LENVIMA + everolimus in DTC, RCC, and HCC, fistula or gastrointestinal perforation occurred in 2%. Permanently discontinue in patients who develop gastrointestinal perforation of any severity or grade 3-4 fistula.

QT Interval Prolongation. In DTC, QT/QTc interval prolongation occurred in 9% of LENVIMA-treated patients and QT interval prolongation of >500 ms occurred in 2%. In RCC, QTc interval increases of >60 ms occurred in 11% of patients receiving LENVIMA + everolimus and QTc interval >500 ms occurred in 6%. In HCC, QTc interval increases of >60 ms occurred in 8% of LENVIMA-treated patients and QTc interval >500 ms occurred in 2%.

Monitor and correct electrolyte abnormalities at baseline and periodically during treatment. Monitor electrocardiograms in patients with congenital long QT syndrome, congestive heart failure, bradyarrhythmias, or those who are taking drugs known to prolong the QT interval, including Class Ia and III antiarrhythmics. Withhold and resume at reduced dose upon recovery based on severity.

Hypocalcemia. In DTC, grade 3-4 hypocalcemia occurred in 9% of LENVIMA-treated patients. In 65% of cases, hypocalcemia improved or resolved following calcium supplementation with or without dose interruption or dose reduction. In RCC, grade 3-4 hypocalcemia occurred in 6% of LENVIMA + everolimus– treated patients. In HCC, grade 3 hypocalcemia occurred in 0.8% of LENVIMA-treated patients. Monitor blood calcium levels at least monthly and replace calcium as necessary during treatment. Withhold and resume at reduced dose upon recovery or permanently discontinue depending on severity.

Reversible Posterior Leukoencephalopathy Syndrome. Across clinical studies of 1,823 patients who received LENVIMA as a single agent, RPLS occurred in 0.3%. Confirm diagnosis of RPLS with MRI. Withhold and resume at reduced dose upon recovery or permanently discontinue depending on severity and persistence of neurologic symptoms.

Hemorrhagic Events. Serious including fatal hemorrhagic events can occur with LENVIMA. In DTC, RCC, and HCC clinical trials, hemorrhagic events, of any grade, occurred in 29% of the 799 patients treated with LENVIMA as a single agent or in combination with everolimus. The most frequently reported hemorrhagic events (all grades and occurring in at least 5% of patients) were epistaxis and hematuria. In DTC, grade 3-5 hemorrhage occurred in 2% of LENVIMA-treated patients, including 1 fatal intracranial hemorrhage among 16 patients who received LENVIMA and had CNS metastases at baseline. In RCC, grade 3-5 hemorrhage occurred in 8% of LENVIMA + everolimus–treated patients, including 1 fatal cerebral hemorrhage. In HCC, grade 3-5 hemorrhage occurred in 5% of LENVIMA-treated patients, including 7 fatal hemorrhagic events. Serious tumor-related bleeds, including fatal hemorrhagic events, occurred in LENVIMA-treated patients in clinical trials and in the postmarketing setting. In postmarketing surveillance, serious and fatal carotid artery hemorrhages were seen more frequently in patients with anaplastic thyroid carcinoma (ATC) than other tumors. Safety and effectiveness of LENVIMA in patients with ATC have not been demonstrated in clinical trials.

Consider the risk of severe or fatal hemorrhage associated with tumor invasion or infiltration of major blood vessels (eg, carotid artery). Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity.

Impairment of Thyroid Stimulating Hormone Suppression/Thyroid Dysfunction. LENVIMA impairs exogenous thyroid suppression. In DTC, 88% of patients had baseline thyroid stimulating hormone (TSH) level ≤0.5 mU/L. In patients with normal TSH at baseline, elevation of TSH level >0.5 mU/L was observed post baseline in 57% of LENVIMA-treated patients. In RCC and HCC, grade 1 or 2 hypothyroidism occurred in 24% of LENVIMA + everolimus–treated patients and 21% of LENVIMA-treated patients, respectively. In patients with normal or low TSH at baseline, elevation of TSH was observed post baseline in 70% of LENVIMA-treated patients in HCC and 60% of LENVIMA + everolimus–treated patients in RCC.

Monitor thyroid function prior to initiation and at least monthly during treatment. Treat hypothyroidism according to standard medical practice.

Wound Healing Complications. Wound healing complications, including fistula formation and wound dehiscence, can occur with LENVIMA. Withhold for at least 6 days prior to scheduled surgery. Resume after surgery based on clinical judgment of adequate wound healing. Permanently discontinue in patients with wound healing complications.

Embryo-fetal Toxicity. Based on its mechanism of action and data from animal reproduction studies, LENVIMA can cause fetal harm when administered to pregnant women. In animal reproduction studies, oral administration of lenvatinib during organogenesis at doses below the recommended clinical doses resulted in embryotoxicity, fetotoxicity, and teratogenicity in rats and rabbits. Advise pregnant women of the potential risk to a fetus; and advise females of reproductive potential to use effective contraception during treatment with LENVIMA and for at least 30 days after the last dose.

Adverse Reactions
In DTC, the most common adverse reactions (≥30%) observed in LENVIMA-treated patients were hypertension (73%), fatigue (67%), diarrhea (67%), arthralgia/myalgia (62%), decreased appetite (54%), decreased weight (51%), nausea (47%), stomatitis (41%), headache (38%), vomiting (36%), proteinuria (34%), palmar-plantar erythrodysesthesia syndrome (32%), abdominal pain (31%), and dysphonia (31%). The most common serious adverse reactions (≥2%) were pneumonia (4%), hypertension (3%), and dehydration (3%). Adverse reactions led to dose reductions in 68% of LENVIMA-treated patients; 18% discontinued LENVIMA. The most common adverse reactions (≥10%) resulting in dose reductions were hypertension (13%), proteinuria (11%), decreased appetite (10%), and diarrhea (10%); the most common adverse reactions (≥1%) resulting in discontinuation of LENVIMA were hypertension (1%) and asthenia (1%).

In RCC, the most common adverse reactions (≥30%) observed in LENVIMA + everolimus–treated patients were diarrhea (81%), fatigue (73%), arthralgia/myalgia (55%), decreased appetite (53%), vomiting (48%), nausea (45%), stomatitis (44%), hypertension (42%), peripheral edema (42%), cough (37%), abdominal pain (37%), dyspnea (35%), rash (35%), decreased weight (34%), hemorrhagic events (32%), and proteinuria (31%). The most common serious adverse reactions (≥5%) were renal failure (11%), dehydration (10%), anemia (6%), thrombocytopenia (5%), diarrhea (5%), vomiting (5%), and dyspnea (5%). Adverse reactions led to dose reductions or interruption in 89% of patients. The most common adverse reactions (≥5%) resulting in dose reductions were diarrhea (21%), fatigue (8%), thrombocytopenia (6%), vomiting (6%), nausea (5%), and proteinuria (5%). Treatment discontinuation due to an adverse reaction occurred in 29% of patients.

In HCC, the most common adverse reactions (≥20%) observed in LENVIMA-treated patients were hypertension (45%), fatigue (44%), diarrhea (39%), decreased appetite (34%), arthralgia/myalgia (31%), decreased weight (31%), abdominal pain (30%), palmar-plantar erythrodysesthesia syndrome (27%), proteinuria (26%), dysphonia (24%), hemorrhagic events (23%), hypothyroidism (21%), and nausea (20%).

The most common serious adverse reactions (≥2%) were hepatic encephalopathy (5%), hepatic failure (3%), ascites (3%), and decreased appetite (2%). Adverse reactions led to dose reductions or interruption in 62% of patients. The most common adverse reactions (≥5%) resulting in dose reductions were fatigue (9%), decreased appetite (8%), diarrhea (8%), proteinuria (7%), hypertension (6%), and palmar-plantar erythrodysesthesia syndrome (5%). Treatment discontinuation due to an adverse reaction occurred in 20% of patients. The most common adverse reactions (≥1%) resulting in discontinuation of LENVIMA were fatigue (1%), hepatic encephalopathy (2%), hyperbilirubinemia (1%), and hepatic failure (1%).

Use in Specific Populations
Because of the potential for serious adverse reactions in breastfed infants, advise women to discontinue breastfeeding during treatment and for at least 1 week after last dose. LENVIMA may impair fertility in males and females of reproductive potential.

No dose adjustment is recommended for patients with mild (CLcr 60-89 mL/min) or moderate (CLcr 30-59 mL/min) renal impairment. LENVIMA concentrations may increase in patients with DTC or RCC and severe (CLcr 15-29 mL/min) renal impairment. Reduce the dose for patients with RCC or DTC and severe renal impairment. There is no recommended dose for patients with HCC and severe renal impairment. LENVIMA has not been studied in patients with end stage renal disease.

No dose adjustment is recommended for patients with HCC and mild hepatic impairment (Child-Pugh A). There is no recommended dose for patients with HCC with moderate (Child-Pugh B) or severe (Child-Pugh C) hepatic impairment.

No dose adjustment is recommended for patients with DTC or RCC and mild or moderate hepatic impairment. LENVIMA concentrations may increase in patients with DTC or RCC and severe hepatic impairment. Reduce the dose for patients with DTC or RCC and severe hepatic impairment.

Please see Prescribing information for LENVIMA (lenvatinib) at View Source

About the Eisai and Merck Strategic Collaboration
In March 2018, Eisai and Merck, known as MSD outside the United States and Canada, through an affiliate, entered into a strategic collaboration for the worldwide co-development and co-commercialization of LENVIMA. Under the agreement, the companies will jointly develop, manufacture and commercialize LENVIMA, both as monotherapy and in combination with Merck’s anti-PD-1 therapy KEYTRUDA.

In addition to ongoing clinical studies evaluating the KEYTRUDA plus LENVIMA combination across several different tumor types, the companies will jointly initiate new clinical studies through the LEAP (LEnvatinib And Pembrolizumab) clinical program, which will evaluate the combination to support 11 potential indications in six types of cancer (endometrial carcinoma, hepatocellular carcinoma, melanoma, non-small cell lung cancer, squamous cell carcinoma of the head and neck, and urothelial cancer). The LEAP clinical program also includes a new basket trial targeting six additional cancer types (biliary tract cancer, breast cancer, colorectal cancer, gastric cancer, glioblastoma and ovarian cancer).

Sirnaomics’ siRNA Therapeutic Candidate, STP705, Granted Orphan Drug Designation By US FDA for Treatment of Hepatocellular Carcinoma

On February 10, 2020 Sirnaomics, Inc., a leading biopharmaceutical company in discovery and development of RNAi therapeutics, reported that the Office of Orphan Product Development division of the FDA has granted orphan drug designation to its leading therapeutic candidate, STP705, for the treatment of Hepatocellular Carcinoma (HCC) (Press release, Sirnaomics, FEB 10, 2020, View Source [SID1234554110]). This is the third such designation for this drug candidate, following the designation for Primary Sclerosing Cholangitis and Cholangiocarcinoma.

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Sirnaomics lead product candidate, STP705 is a siRNA (small interfering RNA) therapeutic that takes advantage of the Company’s proprietary dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expression. Sirnaomics has opened Investigational New Drug (IND) applications under the jurisdiction of the US FDA and Chinese FDA for clinical studies for Non-melanoma skin cancers (NMSC), Cholangiocarcinoma (CCA), and Hypertrophic Scar (HTS) Reduction.

"We are very pleased to reach another significant milestone and very excited to develop our lead product candidate for treatment of HCC with tremendous unmet needs" stated Patrick Lu, PhD, President and CEO. "This third grant of orphan drug designation for STP705 to treat HCC further consolidates our long-term strategy positioning us to take advantage of the regulatory arbitrage by achieving approval in US and then moving to the large market in China."

"Receipt of the orphan drug designation for Hepatocellular Carcinoma is a very important step for Sirnaomics. It provides the pathway allowing us to develop STP705 in a devastating oncology disease for which there is currently no effective therapy," stated Dr. Michael Molyneaux, MD, MBA, Chief Medical Officer. "This orphan drug designation aligns with our mission to alleviate human suffering and target diseases with high unmet clinical need and we anticipate the start of our clinical study for HCC in the second half of 2020."

About STP705 (Cotsiranib)
Sirnaomics leading product candidate, STP705, is an siRNA (small interfering RNA) therapeutic which takes advantage of a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expression. The product candidate has received multiple IND approvals from both the US FDA and Chinese NMPA, including treatments of Cholangiocarcinoma, Non-Melanoma skin cancer and Hypertrophic Scar. STP705 has also received Orphan Drug Designation for treatment of Cholangiocarcinoma and Primary Sclerosing Cholangitis. Preclinical animal models using STP705, have demonstrated a dramatic improvement in T-cell penetration into tumors in the liver with single agent action as well as improvement in the efficacy of an anti-PD-L1 antibody checkpoint inhibitor in an HCC model. This effect may improve other immune checkpoint inhibitor efficacies in addition to those targeting the PD-1/PD-L1 axis.

About Hepatocellular carcinoma (HCC)
Hepatocellular carcinoma (HCC) is the most common type of primary liver cancer in adults, and is the most common cause of death in people with liver cirrhosis. It occurs in the setting of chronic liver inflammation, and is most closely linked to chronic viral hepatitis infection (hepatitis B or C) or exposure to toxins such as alcohol or aflatoxin. Certain diseases, such as hemochromatosis and alpha 1-antitrypsin deficiency, markedly increase the risk of developing HCC. Metabolic syndrome and NASH are also increasingly recognized as risk factors for HCC. Worldwide, approximately 840,000 people are diagnosed with primary liver cancer each year and about 500,000 of the patients are in China, even though HCC is designated as an orphan drug indication by US FDA. Abundant literature has directly linked high level expression of TGF-β1 to liver epithelial to mesenchymal transition (EMT) and tumorigenesis. Inhibition of COX-2 is able to reverse the drug resistance to the chemo drug treatment of HCC and also increased CD4+ T cells within the tumor.

Arvinas Selects Certara to Advance Speed and Efficiency of Its Targeted Protein Degradation Research

On February 10, 2020 Certara, the global model-informed drug development and decision support leader, reported that its discovery research relationship with Arvinas has expanded to include a greater diversity of small molecule drug discovery projects and data sources. Arvinas’ current drug discovery focus areas include metastatic prostate cancer, locally-advanced or metastatic breast cancer, and neurodegenerative diseases (Press release, Arvinas, FEB 10, 2020, View Source [SID1234554109]).

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Arvinas is the pioneer and clinical-stage leader in the exciting new area of targeted protein degradation. Its proprietary PROTAC (proteolysis-targeting chimera) protein degraders are chimeric, modular small molecules, which harness the body’s natural protein disposal system, the ubiquitin‐proteasome system, to induce the degradation of disease‐causing proteins.

"Arvinas is employing Certara’s D360 scientific informatics platform to increase the speed and efficiency of its lead identification and optimization processes. D360 is specifically designed to allow scientific researchers to access, understand and share drug discovery data quickly," explained Dr. David Lowis, Senior Director of Product Management at Certara. "Medical research is moving so rapidly, especially in oncology, that gaining access to novel technology and fast, accurate drug discovery results will determine how quickly new medicines can be brought to patients."

"We need to be able to query, pivot and dive deep into our drug discovery data, and D360 is the best platform we have found for that," said Dr. Keith Hornberger, Director, Chemistry at Arvinas. "We have increased the number of data sources and complexity of data, which are now seamlessly integrated providing complete transparency to our end users. We currently have around 70 scientists using D360, running over 700 distinct D360 queries. Compared to our previous research informatics solution, D360 is saving us hundreds of hours annually on data analysis. That is an important contribution to our research."

"The Certara team is tenacious and resourceful. They always find a solution. That’s what has made our relationship so successful," added Dr. Hornberger.

Designed to meet clients’ specific research goals, D360 features small molecule discovery, biologics discovery, and pre-clinical toolkits for both large and small biopharmaceutical companies.

FerGene to Present Bladder Cancer Data at 2020 ASCO Genitourinary Cancers Symposium

On February 10, 2020 FerGene, a newly created gene therapy company committed to revolutionizing the treatment of bladder cancer, reported it will present research at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium taking place February 13-15 in San Francisco, California (Press release, FerGene, FEB 10, 2020, View Source [SID1234554108]).

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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

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FerGene will present two posters on high-grade, Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC), including an analysis of real-world utilization and outcomes of bladder preservation therapies as well as safety and efficacy results from the Phase 3 study of nadofaragene firadenovec, the company’s investigational intravesical gene therapy in development for patients with high-grade, BCG-unresponsive NMIBC.

Details for the presentations are as follows:

Poster (Abstract 442)

Title: Safety and efficacy of intravesical nadofaragene firadenovec for patients with high-grade, BCG unresponsive nonmuscle invasive bladder cancer (NMIBC): Results from a Phase III Trial

Presented by Stephen A. Boorjian, MD; Professor of Urology, Vice Chair of Research for the Department of Urology, and the Director of the Urologic Oncology Fellowship at Mayo Clinic

Time: Friday, February 14, 2020, 7:20AM – 7:25AM (Rapid Abstract Presentation) and 12:15PM – 1:45PM PST

Poster (Abstract 453)

Title: Disease progression among patients who receive available bladder preservation therapies after failure of BCG therapy in the SEER-Medicare data

Senior Author: Ashish Kamat, MD; Professor of Urology and Director of Urologic Oncology Fellowship at MD Anderson Cancer Center

Time: Friday, February 14, 2020, 12:15PM – 1:45PM PST

About nadofaragene firadenovec

Nadofaragene firadenovec (rAd-IFN/Syn3) is an investigational intravesical 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 for interferon alfa-2b, administered by catheter into the bladder once every three months. The virus is designed to enter 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 production of high and durable quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby uses the patient’s own bladder wall cells to produce interferon, enhancing the body’s natural defenses against the cancer.

A Biologics License Application for nadofaragene firadenovec has been submitted to the U.S. Food and Drug Administration (FDA) for the treatment of patients with high-grade, BCG-unresponsive NMIBC. The application was granted Priority Review, and nadofaragene firadenovec previously received Fast Track and Breakthrough Therapy Designations. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of May 2020.

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.1 It is estimated that there will be approximately 81,000 new cases of bladder cancer in the U.S. in 2020; more than 70% of these cases present as NMIBC.2,3 In patients with high-grade NMIBC, intravesical BCG is the recommended treatment; however, between 30% and 50% of cases with high-grade disease will recur.4 The outcome for BCG-unresponsive patients is poor, with total cystectomy (complete removal of the bladder) often being the recommended next treatment option.5