On October 28, 2015 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported that the independent committee of the National Cancer Institute-Molecular Analysis for Therapy Choice (NCI-MATCH) clinical trial has chosen LOXO-101 as the sole, dedicated treatment arm for patients with tropomyosin receptor kinase, or TRK, gene fusions (Press release, Loxo Oncology, OCT 28, 2015, View Source [SID:1234507816]). The NCI-MATCH trial will initially enroll 3,000 patients with tumor biopsies available for comprehensive genomic profiling and assign these patients to an appropriate targeted therapy arm based on the molecular abnormalities of each tumor. NCI-MATCH, which began enrolling patients in August 2015, currently has 10 open treatment arms employing drugs from multiple sponsors, and plans to open as many as 25 additional arms over the next year. Each arm of the study pairs specific genetic alterations to a specific targeted treatment.

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!

"We are honored that an independent committee of the NCI-MATCH trial has selected LOXO-101 for the TRK fusion arm of the NCI-MATCH trial," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "NCI-MATCH captures the true spirit of precision medicine, and should answer many important questions for patients with advanced cancer. We look forward to working closely with NCI and its national network of investigators."

About NCI-MATCH

NCI-MATCH is supported by NCI and coordinated by the ECOG-ACRIN Cancer Research Group, part of the NCI’s National Clinical Trials Network (NCTN). The NCTN has national reach and NCI-MATCH is opening at NCTN clinical sites across the United States. The trial opened for enrollment in August 2015 at more than 700 clinical trial sites in 48 states with the initial goal of analyzing tumor biopsies from 3,000 patients. Each arm utilizes only one targeted therapy and enrolls adults with advanced of genetically defined solid tumors and lymphomas that are no longer responding (or never responded) to standard therapy and have begun to grow. Additional arms will be added to NCI-MATCH through 2016. The primary endpoint for NCI-MATCH is the objective response rate, or ORR, defined as the percentage of patients whose tumors have a complete or partial response to treatment.

Biopsy specimens removed from patients’ tumors are sent to a central pathology core site at the MD Anderson Cancer Center. Nucleic acids are isolated and assayed for more than 4,000 different variants across 143 genes at one of four clinical genetic testing labs. To efficiently investigate the effectiveness of molecularly targeted therapies for patients with the corresponding driver mutations, a broad-based genomic pre-screening effort such as this is necessary to find and assign patients whose tumors harbor these mutations, regardless of tumor origin. The trial covers the cost of the biopsy and molecular tests, and patients will receive the drugs without charge if they are eligible to enroll for an NCI-MATCH treatment. Neither the patients nor a health plan/insurance company will have to pay for any study-related biopsies or the assigned study drug(s) that were matched to a patient’s cancer.

For more information, see the NCI’s website here. Patients, families, and clinicians can also call NCI’s Cancer Information Service at 1-800-4-CANCER (1-800-422-6237) for assistance in English and Spanish or contact NCI’s LiveHelp service.

About LOXO-101

LOXO-101 is a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities involving the tropomyosin receptor kinases (TRKs). Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In an ongoing Phase 1 clinical trial, LOXO-101 has demonstrated encouraging preliminary efficacy. LOXO-101 is also being evaluated in a global Phase 2 multi-center basket trial in patients with solid tumors that harbor TRK gene fusions. For additional information about both the LOXO-101 clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123.

On October 28, 2015 Ipsen (Euronext: IPN) (ADR: IPSEY) and Telesta Therapeutics Inc. (TSX: TST) (PNK: BNHLF) reported that they have entered into an exclusive licensing agreement for Ipsen to develop and commercialize MCNA1 for the treatment of high risk non-muscle invasive bladder cancer (NMIBC) in all countries of the world, with the exception of the United States, where Telesta is establishing commercial operations, Canada, South Africa, Mexico, South Korea and Japan (Press release, Ipsen, OCT 28, 2015, View Source [SID:1234507815]).

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!

Telesta recently filed a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for MCNA for the treatment of high risk non-muscle invasive bladder cancer patients who are refractory or relapsing from BCG front-line treatment. The FDA has assigned priority review to Telesta’s BLA with a review (PDUFA) date of February 27, 2016. Telesta retains full and sole ownership of MCNA rights in the US and Japan and will be responsible for the commercial launch of MCNA in the United States while Ipsen will initiate discussions with regulatory authorities to identify the regulatory path and potential requirements for the product in Europe and other key licensed territories.

Commenting on this partnership, Dr. Michael Berendt, Chief Executive Officer and Chief Scientist of Telesta Therapeutics noted: "Ipsen is the ideal commercial partner to bring MCNA to patients in the key pharmaceutical markets outside of the United States. They are a recognized development and commercial leader in the field of uro-oncology and are committed to collaborating with our team to ensure that MCNA is brought forward as rapidly as possible to provide a therapeutic option for this underserved patient population. Their extensive knowledge of the regulatory and commercial landscape, their commercial presence in more than 100 countries across the globe, as well as their commitment to their core urology franchise, particularly bladder cancer, is why we are convinced that they will successfully bring MCNA to urologists and their patients, outside of the United States, and generate significant value for Telesta’s shareholders."

Marc de Garidel, Chairman and Chief Executive Officer of Ipsen stated: "Ipsen is pleased to enter into a partnership with Telesta Therapeutics for Europe and key Rest of the World territories. We believe MCNA, which received priority review from FDA, is a promising second line bladder cancer treatment that would perfectly fit our urology-oncology portfolio in Europe." Marc de Garidel added: "This licensing agreement fits our business development strategy, focusing on selected niche therapeutic areas".

Under the financial terms of the agreement, Telesta is eligible to receive up to US$137 million in upfront and milestone payments comprising a US$10 million upfront payment and additional payments contingent upon achievement of regulatory and sales milestones. In addition, Telesta is eligible to receive meaningful tiered double-digit royalties on net sales of MCNA in the licensed territories.

About MCNA

MCNA is a biologic therapy developed to provide high risk non-muscle invasive bladder cancer patients who are refractory to or relapsing from first line therapy with bacillus Calmette-Guérin (BCG), with a therapeutic alternative to surgery. MCNA is derived from the cell wall fractionation of a non-pathogenic bacteria. Its activity is believed to be through a dual mechanism of immune stimulation and direct anti-cancer effects. MCNA was developed to be delivered as a sterile suspension for intravesical administration by urologists and urology nurses, following the same dosing paradigm as first line BCG therapy, with the advantage that it can be prepared, handled and disposed of easily and safely. The efficacy, duration of response and safety data from MCNA’s pivotal Phase 3 trial was recently published2 in The Journal of Urology. The FDA has set February 27, 2016 as its review goal date for MCNA’s potential approval. MCNA offers a new therapeutic option for high risk NMIBC patients and, if approved, will represent the first new therapeutic approved for these patients in the United States since 1989.

About non-muscle invasive bladder cancer (NMIBC)

Treatment options for high risk NMIBC patients who fail first line BCG treatment are extremely limited and treatment guidelines in most countries around the world call for radical cystectomy, which entails a surgical removal of the bladder and adjacent organs and glands. Bladder removal is a complex surgery associated with at least 28% to 45% surgical complications and up to 8% mortality, in addition to negatively impacting multiple aspects of quality of life. Patients who refuse or are not medically fit to undergo bladder removal face an increased risk of progression to muscle-invasive disease, likely leading to metastases and death.