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UroGen Pharma Announces FDA Filing Acceptance and Priority Review of U.S. New Drug Application (NDA) for UGN-101

On December 19, 2019 UroGen Pharma Ltd. (Nasdaq: URGN) reported the U.S. Food and Drug Administration (FDA) accepted for filing and granted priority review for its New Drug Application (NDA) for UGN-101 (mitomycin gel) for instillation as a potential treatment for patients with low-grade upper tract urothelial cancer (LG UTUC) (Press release, UroGen Pharma, DEC 19, 2019, View Source [SID1234552525]). If approved, UGN-101 would be the first non-surgical treatment option for LG UTUC.

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"The FDA filing acceptance and granting of priority review for UGN-101 is an important milestone in our mission to pioneer new treatments to improve patient care in specialty cancers and urologic diseases," said Liz Barrett, President and Chief Executive Officer of UroGen. "There is a significant unmet need for a better treatment option for patients with LG UTUC, as the current standard of care involves surgical removal of the kidney or repetitive endoscopic tumor removal."

The FDA grants priority review to applications for medicines that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Priority review designation shortens the review period from the standard 10 months to six months from the submission of the NDA. The FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of April 18, 2020.

The company is on track for the potential launch of UGN-101 by mid-year 2020. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of LG UTUC.

The NDA is supported by the positive results from the pivotal Phase 3 OLYMPUS clinical trial. Results from a final analysis of the primary endpoint showed that UGN-101 demonstrated a complete response rate of 59 percent in patients with LG UTUC. In addition, the durability of response was estimated as 89 percent at six months and 84 percent at 12 months by Kaplan Meier analysis. Median time to recurrence was estimated to be 13 months. The most commonly reported treatment emergent adverse events were ureteric stenosis (43.7%), urinary tract infection (32.4%), haematuria (31.0%), flank pain (29.6%) nausea (23.9%), dysuria (21.1%), renal impairment (19.7%) and vomiting (19.7%). The majority of these adverse events were mild to moderate, with 8.5% of patients having events of ureteric stenosis reported as severe.

About The Phase 3 OLYMPUS Trial

OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is a pivotal, open-label, single-arm Phase 3 clinical trial of UGN-101 (mitomycin gel) for instillation to evaluate the safety, tolerability and tumor ablative effect of UGN-101 in patients with low-grade UTUC. The trial enrolled 74 patients at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of UGN-101 administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine complete response (CR), the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer. Patients who achieved a CR at the PDE timepoint were then followed for up to 12 months to determine the durability of response with UGN-101.

About UGN-101

UGN-101 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard ureteral catheters. The Company initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the treatment of LG UTUC.

Turnstone Biologics Announces Global Collaboration and License Agreement with Takeda to Develop Novel Viral Immunotherapies

On December 19, 2019 Turnstone Biologics, a biotechnology company pioneering the development of engineered viral immunotherapies, reported a strategic collaboration with Takeda Pharmaceutical Company Limited ("Takeda") to develop multiple products from its proprietary vaccinia virus platform targeting a broad range of cancer indications (Press release, Turnstone Biologics, DEC 19, 2019, View Source [SID1234552524]). The parties will advance Turnstone’s lead program, RIVAL-01, through a worldwide co-development and co-commercialization partnership and will also conduct collaborative discovery efforts to identify additional novel product candidates based on the vaccinia virus platform for future independent development.

"Our collaboration with Takeda will combine our exciting viral immunotherapy platform with Takeda’s deep immuno-oncology research development expertise and proprietary technologies to discover and advance new medicines that have the potential to address critical gaps in the treatment of cancer that exist today," said Sammy Farah, Ph.D., CEO and President, Turnstone Biologics. "Importantly, this partnership allows us to co-develop and co-commercialize RIVAL-01 together with Takeda, enabling us to broaden our internal capabilities and expand our viral immunotherapy pipeline, while retaining our ability to independently develop other candidates based on this technology."

Under the terms of the agreement, Turnstone will receive a total of $120 million in upfront cash, near-term milestones and future equity investment. The collaboration agreement grants Takeda an exclusive worldwide license to co-develop and co-commercialize RIVAL-01 with Turnstone, with global costs and profits shared 50:50. The companies will also collaborate on the development of new product candidates based on Turnstone’s proprietary vaccinia virus platform. Takeda has the right to license select candidates resulting from the collaboration, with Turnstone retaining ownership of the others to advance independently. Turnstone is eligible to receive up to an additional $900 million in potential development, regulatory and commercial milestones across all programs, and receive royalty payments on net sales of each licensed product.

"Our immuno-oncology discovery engine is focused on novel, differentiated mechanisms throughout the cancer immunity cycle and we are privileged to add engineered viral immunotherapies to our portfolio," said Chris Arendt, Head, Oncology Drug Discovery Unit at Takeda. "Our partnership with Turnstone and its vaccinia virus platform will help us harness the power of the immune system in unique ways to address some of the most difficult-to-treat cancers."

Turnstone’s RIVAL therapeutic pipeline is based on its proprietary vaccinia virus platform, which has been engineered for enhanced immune-stimulation and tumor cell selectivity, potent oncolysis and large transgene carrying capacity. RIVAL-01 is the lead candidate, consisting of the vaccinia virus backbone encoding transgenes for Flt3 ligand, anti-CTLA-4 antibody and IL-12 cytokine. The transgenes are designed to be expressed when the vaccinia virus enters and replicates in cancer cells throughout the body. The resulting local production of these therapeutics at the site of tumors adds to the inherent oncolytic and microenvironment-modifying properties of the virus to form a powerful multi-modal attack on the disease.

"Our proprietary vaccinia virus platform is exquisitely engineered to enhance virus-mediated cancer cell killing and better harness the power of the immune system against tumors, with the aim of transforming the treatment paradigm and developing much-needed therapies for people with cancer," said Mike Burgess, Ph.D., President of R&D, Turnstone Biologics. "With RIVAL-01, we intend to deliver three powerful immune modulating agents to primary and metastatic tumor sites and limit their expression to the local tumor environment, reducing the potential for systemic toxicity. This therapy has the potential to drive immune activity in the tumor that is not otherwise achievable."

Median Technologies Announces the Signing of a € 35 Million Finance Contract with the European Investment Bank (EIB)

On December 19, 2019 Median Technologies (Paris:ALMDT), The Imaging Phenomics Company (Euronext Growth – ALMDT) and the European Investment Bank (EIB) reported the signing of a € 35 million finance contract, supported by the European Strategic Investment Fund (EFSI) or "Juncker Plan" (Press release, MEDIAN Technologies, DEC 19, 2019, View Source [SID1234552517]). News about ongoing negotiations for this loan was previously released on May 15, 2019.

This financing, divided into three (3) tranches, will enable Median Technologies to enhance and accelerate its iBiopsy imaging platform investment program for the coming years. Median is a cutting-edge AI and data sciences technology provider for precision medicine. Through its proprietary iBiopsy platform, Median is developing non-invasive imaging biomarkers to enable the identification of certain chronic disease – including cancer- signatures, to dramatically enhance early detection, severity quantification and monitoring of diseases. The objective is on one hand to guide clinicians in their therapeutic decisions in the context of precision and predictive medicine and, on the other hand, to provide disruptive decision tools to foster medical innovations and new therapy development.

Median will request the disbursement of the first tranche of € 15 million during the first semester of 2020. The contract then provides for the disbursement of the second and third tranches (of € 10 million each) in the coming years, at Median Technologies’ discretion, subject to the completion of certain conditions precedents specified in the finance contract. The repayment of this financing will occur, in a single installment, at the end of a five -year period after the disbursement date. The finance contract is supplemented by the payment of various interests and fees and by a guarantee granted by Median Technologies, Inc. (Median Technologies’ US subsidiary).

Pursuant to the warrants issuance agreement, Median Technologies will issue 800,000 warrants for the benefit of the EIB on the date of disbursement of the first tranche and, where appropriate, 300,000 additional warrants on the date of disbursement of the second tranche, at a subscription price of € 0.01. The exercise price of these warrants will be determined based on the price of one or more fundraising(s) of at least € 15 million carried out within 15 months after the subscription date to which an increasing discount will apply based on time, with a minimum of € 2 from the 16th month. The lifespan of these warrants is 15 years.

The warrants issuance agreement includes an exercise parity adjustment clause which could apply, under certain conditions,in case of capital increase. The EIB will be granted with the possibility, under certain conditions, to request Median Technologies to buy back its warrants for a maximum amount of € 50 million and, beyond that amount, to find a buyer and pay interests on the price of the remaining warrants. The total amount of warrants (for the two tranches) would represent up to 7.44 % of the share capital fully diluted.

The objective of this financing, granted by the EIB together with the European guarantee within the framework of the Juncker plan, is to support research and innovation projects developed by companies with substantial growth potential. Median Technologies meets these criteria as its technologies have the potential to impact the lives of hundreds of thousands of patients worldwide.

The agreement was signed by the European Investment Bank and Fredrik Brag, CEO and co-founder of Median Technologies on December 18, 2019.

"Through the Juncker Plan impulse, EIB has become a key player in financing innovative companies, in particular companies involved in the domains of Health and Artificial intelligence, which are the core activities developed by Median Technologies ", said EIB Vice-President, Mr. Ambroise Fayolle

Fredrik Brag, CEO and co-founder of Median Technologies added: "We are very pleased to announce the signing of the finance agreement with the European Investment Bank. The EIB financing will allow us to accelerate our investment in the development of our iBiopsy platform with a strong focus in oncology and liver disease. We are the next generation precision medicine company focused on helping conquer cancer and other diseases through our proprietary routine imaging tests. These novel non-invasive imaging tests could dramatically impact early detection, diagnosis and monitoring of diseases. We leverage our capabilities in technology, artificial intelligence, clinical development, regulatory and reimbursement to drive the development and commercial adoption of our future iBiopsy product line, improve patient clinical outcome and lower healthcare costs."

PharmaMar and Jazz Pharmaceuticals Sign Exclusive License Agreement for Lurbinectedin in the U.S.

On December 19, 2019 PharmaMar (MSE:PHM) and Jazz Pharmaceuticals plc (Nasdaq:JAZZ) reported that PharmaMar and Jazz Pharmaceuticals Ireland Limited have entered into an exclusive license agreement for lurbinectedin in the United States (Press release, PharmaMar, DEC 19, 2019, View Source [SID1234552516]).

Under the terms of this agreement, PharmaMar will receive an upfront payment of $200 million with potential regulatory milestone payments of up to $250 million upon the achievement of accelerated and/or full regulatory approval of lurbinectedin by FDA within certain timelines.

PharmaMar is also eligible to receive up to $550 million in potential commercial milestone payments, as well as incremental tiered royalties on future net sales of lurbinectedin ranging from the high teens up to 30 percent. PharmaMar may receive additional payments on approval of other indications. PharmaMar retains production rights for lurbinectedin and will supply the product to Jazz.

Lurbinectedin was granted orphan drug designation for SCLC by FDA in August 2018. In December 2019, PharmaMar submitted an NDA to FDA for accelerated approval of lurbinectedin for relapsed SCLC, based on data from its Phase 2 basket trial, following positive interactions with FDA.

The lurbinectedin Phase 2 monotherapy basket trial enrolled a total of 105 patients at 39 centers in eight Western European countries in addition to the U.S. The primary endpoint was Overall Response Rate (ORR) as measured by investigator review assessment. Secondary endpoints included Duration of Response, Progression-Free Survival, Overall Survival, and safety. In relapsed SCLC, lurbinectedin showed an ORR of 35.2%, which compares favorably to topotecan’s historical ORR of 16.9%1 by investigator assessment. In addition, lurbinectedin demonstrated a favorable safety, tolerability and administration profile versus historical standard of care.

"We are very pleased with the lurbinectedin agreement with our new U.S. partner Jazz," said José María Fernández Sousa-Faro, PhD, President of PharmaMar. "We are convinced that with Jazz, we have found a partner deeply committed to providing lurbinectedin to patients in the U.S. Lurbinectedin has the potential to become a therapeutic alternative for patients with relapsed small cell lung cancer, who have limited treatment options."

"Lurbinectedin represents a strong strategic fit and an exciting opportunity for Jazz to expand our oncology portfolio with a late stage asset," said Bruce C. Cozadd, Chairman and CEO of Jazz Pharmaceuticals. "We are looking forward to commercializing lurbinectedin in the U.S., as SCLC is an area of significant unmet medical need given limited late-stage treatment options and we believe lurbinectedin may offer patients with relapsed SCLC a meaningful treatment option."

SCLC is a very aggressive cancer that usually is diagnosed with advanced, often metastatic, disease, thus limiting the role of traditional approaches and often posing a worse prognosis when compared to other lung cancers2. In the U.S., approximately 10-15% of lung cancers are small cell.2 Approximately 30,000 new cases of SCLC are recorded in the U.S. every year3.

Closing of the agreement is subject to expiration or termination of the waiting period under the Hart-Scott-Rodino Act.

PharmaMar Conference Call for Investors and Analysts

PharmaMar management will host a conference call and webcast for investors and analysts on January 9th, 2020, at 14:00 CET (08:00 AM, New York time) as follows. The numbers to connect to the teleconference are 877-407-3102 (from USA or Canada) and +1 201-493-6790 (other countries). Interested parties can also follow the conference call live via the following link: https://78449.themediaframe.com/dataconf/productusers/phm/mediaframe/33803/indexl.html

The recording of the teleconference will be available for thirty days and it can be accessed on PharmaMar’s website by visiting the Events Calendar section of the Company’s website www.pharmamar.com.

Jazz Pharmaceuticals Conference Call for Investors and Analysts

Jazz Pharmaceuticals will host an investor conference call and live audio webcast on Friday, January 10, 2020 at 8:30 a.m. EST (1:30 p.m. GMT) to discuss the transaction. The live webcast may be accessed from the Investors section of Jazz Pharmaceuticals’ website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 4069667.

A replay of the conference call will be available through January 17, 2020 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 4069667. An archived version of the webcast will be available for at least one week in the Investors section of Jazz Pharmaceuticals’ website at www.jazzpharmaceuticals.com

About Lurbinectedin
Lurbinectedin (PM1183) is a synthetic compound currently under clinical investigation. It is a selective inhibitor of the oncogenic transcription programs on which many tumors are particularly dependent. Together with its effect on cancer cells, lurbinectedin inhibits oncogenic transcription in tumor-associated macrophages, downregulating the production of cytokines that are essential for the growth of the tumor. Transcriptional addiction is an acknowledged target in those diseases, many of them lacking other actionable targets.

OPKO Health to Present at the 38th Annual J.P. Morgan Healthcare Conference

On December 19, 2019 OPKO Health, Inc. (OPK) reported that management will be participating in the 38th Annual J.P. Morgan Healthcare Conference being held January 13-16, 2020 at the Westin St. Francis in San Francisco (Press release, Opko Health, DEC 19, 2019, View Source [SID1234552515]). Management will be hosting one-on-one meetings with investors and will be presenting on Wednesday, January 15 at 10:30 a.m. Pacific time (1:30 p.m. Eastern time).

The presentation will be webcast live and available for replay in the Investors section of OPKO Health’s website and accessible by clicking here.