On September 4, 2019 DURECT Corporation (Nasdaq: DRRX) reported that James E. Brown, Chief Executive Officer will be presenting at the 21st Annual Global Rodman and Renshaw Investment Conference, sponsored by H. C. Wainwright, at the Lotte New York Palace Hotel in New York on Monday, September 9, 2019 at 11:40 a.m. EDT. Institutional investors and analysts that are attending the conference may request a one-on-one meeting through the conference coordinators (Press release, DURECT, SEP 4, 2019, https://investors.durect.com/news-releases/news-release-details/durect-present-21st-annual-rodman-and-renshaw-global-investment?field_nir_news_date_value[min]=2019 [SID1234539272]).

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A live audio webcast of the presentation will be available by accessing View Source

The live audio webcast of the presentation will also be available by accessing DURECT’s homepage at www.durect.com and clicking on the "Investors" tab. If you are unable to participate during the live webcast, the call will be archived on DURECT’s website in the "Event Calendar" of the "Investors" section.

On September 4, 2019 Leap Therapeutics, Inc. (NASDAQ: LPTX), a biotechnology company developing targeted and immuno-oncology therapeutics, reported that Christopher K. Mirabelli, Ph.D., Chairman, President and Chief Executive Officer, will present a corporate overview at the following investor conferences (Press release, Leap Therapeutics, SEP 4, 2019, View Source [SID1234539289]):

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The H.C. Wainwright 2019 Global Investment Conference in New York City on Monday, September 9, 2019 at 8:20 a.m. ET.
The Ladenburg Thalmann 2019 Healthcare Conference in New York City on Tuesday, September 24, 2019 at 2:00 p.m. ET.
The presentations will be webcast live and may be accessed on the Investors page of the company’s website at www.investors.leaptx.com, where a replay of the events will also be available for a limited time.

On September 4, 2019 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported the launch of Rozlytrek capsules 100 mg and 200 mg (generic name: entrectinib) (hereafter, Rozlytrek), an anticancer agent/tyrosine kinase inhibitor, for the treatment of NTRK fusion-positive advanced or recurrent solid tumors (Press release, Chugai, SEP 4, 2019, View Source [SID1234539257]). Rozlytrek received manufacturing and marketing approval on June 18, 2019 and was listed on the National Health Insurance (NHI) reimbursement price list on September 4.

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"We are very pleased that we can now provide Rozlytrek to patients as the first drug for the treatment of NTRK fusion-positive solid tumors in Japan," said Dr. Osamu Okuda, Chugai’s Executive Vice President, Co-Head of Project & Lifecycle Management Unit. "Rozlytrek is a drug that embodies advanced personalized healthcare as it has been approved to treat solid tumors with rare gene fusions regardless of their site of origin. We will continue to take every measures to provide proper information to ensure the safe use of Rozlytrek."

Rozlytrek is a drug that will realize the advance in personalized healthcare, which is one of the goals set by Chugai. After receiving Sakigake designation from the Ministry of Health, Labour and Welfare (MHLW) for NTRK fusion-positive advanced/recurrent solid tumors with extremely rare gene mutation, Chugai obtained the world’s first regulatory approval for Rozlytrek in Japan on June 18, 2019. Rozlytrek is the first drug approved in Japan for the treatment of NTRK fusion-positive solid tumors as a tumor agnostic treatment in both adult and pediatric patients. In other countries, Rozlytrek has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) and was approved for the treatment of NTRK fusion-positive solid tumors on August 15, 2019. The European Medicines Agency (EMA) has designated Rozlytrek as PRIority MEdicines (PRIME).

For Biomarker testing of NTRK gene fusions, Chugai obtained approval of FoundationOne CDx Cancer Genomic Profile as a companion diagnostic for Rozlytrek by the MHLW on June 26, 2019.

As a leading company in the field of oncology, Chugai will regard Rozlytrek as the drug to realize advanced personalized healthcare in oncology and contribute to patients and healthcare professionals through the promotion of its appropriate use.

[Reference information]
Media release issued by Chugai on June 18, 2019
Title: Anti-Cancer Agent Rozlytrek, Approved for the Treatment of NTRK Fusion Gene Positive Advanced/Recurrent Solid Tumors View Source

Media release issued by Roche on August 16, 2019
Title: FDA approves Roche’s Rozlytrek (entrectinib) for people with ROS1-positive, metastatic non-small cell lung cancer and NTRK gene fusion-positive solid tumours
View Source

Drug Information

Product name:
Rozlytrek Capsules 100 mg
Rozlytrek Capsules 200 mg

Nonproprietary name: Entrectinib

Indications: Neurotrophic tyrosine receptor kinase (NTRK) fusion-positive advanced or recurrent solid tumors

Dosage and administration:
The usual adult dosage is 600 mg entrectinib administered orally once a day. Reduce the dose as necessary depending on the patient’s condition.
The usual pediatric dosage is 300 mg/m2 (body surface area) entrectinib administered orally once a day. However, the dose should not exceed 600 mg. Reduce the dose as necessary depending on the patient’s condition.

Dose for pediatric patients (300 mg/m2 administered orally once a day)

Body surface area (m2) Dose (once a day)
0.43-0.50 100 mg
0.51-0.80 200 mg
0.81-1.10 300 mg
1.11-1.50 400 mg
≧1.51 600 mg
Date of approval: June 18, 2019

Date of NHI reimbursement price listing: September 4, 2019

Date of launch: September 4, 2019

Shelf life: 24 months

Approval conditions:
1. A drug risk management plan is to be prepared and appropriately implemented.
2. Given that the number of patients in clinical studies in Japan was extremely limited, postmarketing drug use surveillance of all patients receiving ROZLYTREK should be conducted until data for a certain number of patients have been accumulated, in order to understand background information on patients receiving ROZLYTREK, collect early data on the safety and efficacy of ROZLYTREK, and take necessary measures for appropriate use of ROZLYTREK.

Drug price:
Rozlytrek Capsules 100 mg JPY 5,214.20/Capsule
Rozlytrek Capsules 200 mg JPY 9,889.90/Capsule

On September 4, 2019 Ribon Therapeutics, a biotechnology company developing first-in-class therapeutics targeting novel enzyme families activated under cellular stress conditions, reported the dosing of the first patient in its Phase 1 clinical trial of RBN-2397, the company’s first-in-class PARP7 inhibitor (Press release, Ribon Therapeutics, SEP 4, 2019, View Source [SID1234539273]). PARP7 belongs to a family of enzymes activated under cellular stress conditions, known as monoPARPs, which are distinct from polyPARPs such as PARP1/2. Recent science pioneered by Ribon has shown monoPARPs are key regulators of stress responses and are linked to the development of certain diseases, including cancers. The Phase 1 trial of RBN-2397 is the first-ever clinical evaluation of a monoPARP inhibitor.

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"With RBN-2397, we are targeting PARP7, which plays a critical role in cancer cell survival. Inhibiting PARP7 has been shown to potently inhibit cancer cell proliferation and also release a ‘brake’ that cancer cells use to evade the immune system," said Sudha Parasuraman, M.D., Chief Medical Officer, Ribon Therapeutics. "We have seen very strong preclinical evidence supporting the clinical potential of RBN-2397, and we believe it can become a valuable new treatment option for cancer patients."

"Bringing our lead compound into the clinic is a key milestone for Ribon, demonstrating our ability to advance from program initiation to the clinic in under three years," said Victoria Richon, Ph.D., President and Chief Executive Officer, Ribon Therapeutics. "This progress validates the utility and efficiency of our platform in rapidly interrogating novel biological pathways and developing clinical drug candidates. The platform has broad potential applications, enabling us to develop first-in-class therapeutics against an expanding set of novel targets in oncology, neurodegeneration and inflammatory disease."

The Phase 1 clinical trial is a multi-center, open-label, dose-escalation study designed to assess the safety, tolerability, and pharmacokinetics of RBN-2397, as well as initial signs of anti-tumor activity in patients with advanced-stage solid tumors. The goals of the study are to establish the recommended dose for future clinical investigation and to explore pharmacodynamic readouts and predictive biomarkers. The study is comprised of a dose escalation phase, which will be followed by a number of expansion cohorts evaluating RBN-2397 in patients with various tumor types, including squamous cell carcinoma of the lung, in which PARP7 has been shown to be genetically amplified. Additional information on this clinical trial can be found on www.clinicaltrials.gov.

RBN-2397 – Inhibiting PARP7, a Key monoPARP Cancer Dependency

Ribon’s lead program, RBN-2397, is focused on inhibiting overactive PARP7 in tumors, which has been shown to play a key role in cancer survival. Ribon’s research has discovered that many cancer cells rely on PARP7 for intrinsic cell survival, and that PARP7 allows cancer cells to "hide" from the immune system. Ribon has demonstrated that inhibition of PARP7 with RBN-2397 can potently inhibit the growth of cancer cells and restore interferon signaling, effectively releasing the "brake" cancer uses to hide from the immune system and suppress both innate and adaptive immune mechanisms. In several cancer models, RBN-2397 demonstrated durable tumor growth inhibition, potent antiproliferative activity and restoration of interferon signaling. Ribon plans to initially develop RBN-2397 in squamous cell carcinoma of the lung, where research has shown PARP7 to be genetically amplified. The company also plans to explore RBN-2397 for the treatment of additional cancers, including cancers of the aerodigestive tract, pancreatic cancer and ovarian cancer.

PARP7 is a member of the monoPARP family of proteins, which are key regulators of stress responses that enable cancer cells to survive and also evade immune detection, and emerging science has linked their activity with disease development. MonoPARPs are a family of 12 enzymes that are functionally and structurally distinct from the more well-known polyPARPs, such as PARP1/2. MonoPARPs function across a variety of stress responses relevant to disease development in cancer, inflammatory conditions and neurodegenerative diseases. Ribon has built an integrated technology platform to interrogate monoPARPs to develop first-in-class, small molecule therapeutics.

On September 4, 2019 Prescient Metabiomics, a privately held company and a subsidiary of Prescient Medicine Holdings, Inc., announced that the U.S. Food and Drug Administration (FDA) has granted the company’s submission for Breakthrough Device designation to its LifeKit Prevent Colorectal Neoplasia Test (Press release, Prescient Metabiomics, SEP 4, 2019, View Source [SID1234539290]). This Breakthrough Device designation is the first designation for a non-invasive diagnostic test that is designed to detect pre-cancerous polyps, as well as early-stage carcinomas, with the potential to aid in the interception of carcinogenesis and prevention of colorectal cancer (CRC). In contrast, other non-invasive tests on the market primarily detect full-blown cancer, not precancerous adenomas, and require programs that rely heavily on colonoscopies to achieve colon cancer prevention.

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The LifeKit Prevent Test deploys patented metagenomics technologies intended to identify microbial DNA and RNA biomarkers that are associated with neoplasia in the lower gastrointestinal tract. Using a non-invasive collection swab, the microbial biomarkers in the stool are analyzed. A positive result indicates the presence of colorectal adenomas or CRC, which should be followed by a diagnostic colonoscopy and polypectomy as appropriate.

A "Breakthrough Device" is an important designation by the FDA because it expedites the review process for medical devices that meet the designation criteria and offers more effective diagnosis of life-threatening diseases and conditions. The Breakthrough Device Program (BDP) offers manufacturers an opportunity to engage with the FDA’s experts to efficiently address topics during the pre-market review phase. Prescient Metabiomics anticipates that FDA support through the BDP will greatly reduce the regulatory timeline required for completing clinical trials and achieving FDA clearance.

"We are excited to deliver LifeKit Prevent, a non-invasive diagnostic test that not only will aid in detecting CRC, but also help identify patients at risk for advanced adenomas. By identifying patients most at risk for precancerous lesions, we can stop cancer before it starts and save lives," says Keri Donaldson, MD, and Chief Executive Officer of Prescient Medicine Holdings, Inc. "This important innovation, developed on our robust R&D and machine learning platforms, will allow more targeted and effective treatments earlier. The FDA’s decision confirms the unique aspects of our technology and the potential benefit of our test in addressing the large and increasing population of people at risk of developing CRC."

About Colon Cancer and LifeKit Prevent

Colorectal cancer (CRC) is the third-most common type of cancer and the second leading cause of cancer death in the United States, with over 137,000 new cases and 56,000 deaths annually. The U.S. spends $14 billion each year for the diagnosis and treatment of CRC with costs largely due to delayed detection. While 14 million colonoscopies are performed annually, two-thirds of Americans do not follow screening guidelines and 60 percent of CRC cases are not detected until later stages. Using non-invasive tests to augment colonoscopy has been shown to increase adherence to CRC screening programs. Estimates suggest that broader CRC screening could save 18,800 lives in the US annually.

Previously completed clinical research indicates that LifeKit Prevent may be more convenient for patients and healthcare providers to use than currently available CRC diagnostic tests. Based on current findings, LifeKit Prevent is highly accurate for detecting colon cancer and, more importantly, for detecting precancerous adenomas that are missed by existing non-invasive tests. A positive result from LifeKit Prevent will provide a stronger case to motivate reluctant, high-risk patients to undergo a colonoscopy resulting in the removal of precancerous adenomas to prevent further progression to carcinogenesis and reduce the morbidity and mortality of CRC.