On May 23, 2019 The partner of CStone Pharmaceuticals ("Cstone"; HKEX:2616), Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) ("Agios") reported that the global Phase 3 ClarIDHy trial of TIBSOVO (ivosidenib) in previously treated cholangiocarcinoma patients with an isocitrate dehydrogenase 1 (IDH1) mutation met its primary endpoint (Press release, CStone Pharmaceauticals, MAY 23, 2019, View Source [SID1234536563]). Treatment with TIBSOVO demonstrated a statistically significant improvement in progression-free survival (PFS) by independent radiology review compared with patients who received placebo. The safety profile observed in the study was consistent with previously published data.

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A full analysis of the ClarIDHy trial will be submitted for presentation at the European Society for Medical Oncology Congress in 2019. Agios plans to submit a supplemental new drug application for TIBSOVO in previously treated IDH1 mutant cholangiocarcinoma by the end of 2019.

Cholangiocarcinoma is a very aggressive tumor for which surgical resection is the primary treatment option. Many patients are undiagnosed until the mid-to-late stages when they have missed the time window for surgery. At present, there is no standard second- and third-line treatment for patients with advanced cholangiocarcinoma. Patients typically suffer a poor prognosis and short-term survival.

China is among the countries with the highest incidence of Cholangiocarcinoma which is associated with a variety of high-risk factors including hepatitis B and Clonorchis sinensis infection, diseases that are endemic in the country.

In June 2018, CStone entered into an exclusive collaboration and license agreement with Agios to develop and commercialize TIBSOVO in Greater China region.

ClarIDHy Phase 3 Trial
The ClarIDHy trial is a global, randomized Phase 3 trial in previously treated IDH1 mutant cholangiocarcinoma patients who have documented disease progression following one or two systemic therapies in the advanced setting. As of the January 31, 2019 data cutoff, 185 patients were randomized.

Patients were randomized 2:1 to receive either single-agent TIBSOVO 500 mg once daily or placebo with crossover to TIBSOVO permitted at the time of documented radiographic progression per RECIST 1.1.
The primary endpoint of the trial is PFS as evaluated by independent radiology review with secondary endpoints including investigator evaluated PFS, safety and tolerability, overall response rate, overall survival, duration of response, PK/PD and quality of life assessments.
The study was designed with 96% power to detect a hazard ratio of 0.5 for PFS (TIBSOVO vs. placebo), with a one-sided alpha of 0.025.
Thermo Fisher Scientific is providing next-generation sequencing to detect IDH1 mutations for all tumor samples as inclusion criteria for enrollment in the study and will develop and commercialize the validated companion diagnostic.
TIBSOVO is not approved in any country for the treatment of patients with advanced cholangiocarcinoma.

About TIBSOVO (ivosidenib)

TIBSOVO is indicated in the United States for the treatment of acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in:

Adult patients with newly-diagnosed AML who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Adult patients with relapsed or refractory AML.

On May 23, 2019 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that it will webcast its presentation at the Goldman Sachs 40th Annual Global Healthcare Conference at 9:20 a.m. Pacific Time (12:20 p.m. Eastern Time) on Tuesday, June 11, 2019 (Press release, Regeneron, MAY 23, 2019, View Source [SID1234536562]).

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The session may be accessed from the "Investors & Media" page of Regeneron’s website at View Source A replay of the webcast will be archived on the Company’s website and will be available for 30 days.

On May 23, 2019 Zymeworks Inc. (NYSE/TSX: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported that it will host a webcast and conference call to discuss progress on its 2019 corporate priorities, including clinical development plans for its lead therapeutic candidates as well as recently announced and established corporate partnerships (Press release, Zymeworks, MAY 23, 2019, View Source [SID1234536559]).

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Webcast and Conference Call Details

Zymeworks will host a webcast and conference call on Wednesday, May 29, 2019 at 8:30am ET (5:30am PT).

Interested parties can access the live webcast via a link from Zymeworks’ website at View Source or join the live call and Q&A by dialing 1-800-319-4610 for North American callers, or 1-604-638-5340 for international callers. Callers should dial in five to 10 minutes prior to the scheduled start time and ask to join the "Zymeworks call".

A recorded replay will also be available on the website shortly after the call concludes.

On May 23, 2019 Novocure (NASDAQ: NVCR) reported that the U.S. Food and Drug Administration (FDA) has approved the NovoTTF-100L System in combination with pemetrexed plus platinum-based chemotherapy for the first-line treatment of unresectable, locally advanced or metastatic, malignant pleural mesothelioma (MPM) (Press release, NovoCure, MAY 23, 2019, View Source [SID1234536558]). NovoTTF-100L is a non-invasive, antimitotic cancer treatment that delivers Tumor Treating Fields to the region of the tumor. Tumor Treating Fields therapy uses electric fields tuned to specific frequencies to disrupt solid tumor cancer cell division.

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NovoTTF-100L is the first treatment for MPM approved by the FDA in more than 15 years. Preclinical data showed that human mesothelioma cells are highly sensitive to Tumor Treating Fields. In the STELLAR registration trial, 80 unresectable MPM patients treated with Tumor Treating Fields plus chemotherapy experienced a median overall survival of 18.2 months (95% CI 12.1-25.8).

MPM is a rare cancer that has been strongly linked to asbestos exposure. Approximately 3,000 people are diagnosed with MPM in the United States annually. Prior to the FDA approval of NovoTTF-100L, pemetrexed plus cisplatin was the only FDA-approved therapy for patients with unresectable MPM.

NovoTTF-100L for MPM is classified as a Humanitarian Use Device (HUD) and was approved under Humanitarian Device Exemption (HDE). The HDE pathway was created to encourage companies to innovate in rare diseases with underserved patient populations. The FDA approved Optune, another Tumor Treating Fields delivery system, under the Premarket Authorization (PMA) pathway in 2011 for the treatment of glioblastoma (GBM). Since 2011, more than 12,000 patients with GBM have been treated with Tumor Treating Fields.

"Since 2000, we have been developing and commercializing Tumor Treating Fields to extend survivals in some of the most aggressive forms of cancer," said Bill Doyle, Novocure’s Executive Chairman. "FDA approval of NovoTTF-100L provides patients with the first FDA-approved treatment for MPM in more than 15 years and, as our first FDA-approved torso cancer indication, marks a major milestone for Novocure. We are thankful for the patients, caregivers and health care providers who partnered with us to make this possible."

"MPM is a devastating disease, with only 10 to 20 percent of patients being candidates for surgery to remove the tumor," said Mary Hesdorffer, NP, Executive Director of the Mesothelioma Applied Research Foundation. "Typically, mesothelioma patients who cannot have surgery receive palliative care to mitigate their symptoms. NovoTTF-100L provides unresectable MPM patients with a treatment option that may improve survival. We are encouraged by the FDA approval and hope it is just the beginning of innovation in the treatment of this aggressive disease."

Efficacy outcomes in the STELLAR trial

The FDA approval is based on the results of the STELLAR trial. STELLAR was a prospective, single-arm trial designed to study the safety and efficacy of NovoTTF-100L plus chemotherapy first-line in patients with unresectable MPM. The trial included 80 patients with unresectable and previously untreated MPM who were candidates for treatment with pemetrexed and cisplatin or carboplatin. The trial was powered to prospectively determine the overall survival in patients treated with NovoTTF-100L plus chemotherapy. Secondary endpoints included overall response rate (per mRECIST criteria), progression free survival and safety.

The median overall survival was 18.2 months (95% CI 12.1-25.8) across all patients treated with NovoTTF-100L plus chemotherapy. The median overall survival was 21.2 months for patients with epithelioid MPM (n=53) and 12.1 months for patients with non-epithelioid MPM (n=21). More than half, 62 percent, of patients (n=80) enrolled in the STELLAR trial who used NovoTTF-100L plus chemotherapy were still alive at one year. The disease control rate in patients with at least one follow-up CT scan performed (n=72) was 97 percent. 40 percent of patients had a partial response, 57 percent had stable disease, and 3 percent had progressive disease. The median progression free survival was 7.6 months.

In addition, the STELLAR trial demonstrated that NovoTTF-100L could be combined with chemotherapy. There was no increase in serious systemic adverse events when NovoTTF-100L was added to chemotherapy. Mild-to-moderate skin irritation was the most common device-related side effect with NovoTTF-100L.

Caution: Federal law restricts this device to sale by or on the order of a physician. Humanitarian Device. Authorized by Federal Law for use in the treatment of adult patients with unresectable, locally advanced or metastatic, malignant pleural mesothelioma concurrently with pemetrexed and platinum based chemotherapy. The effectiveness of this device for this use has not been demonstrated.

On May 23, 2019 OncoNano Medicine, Inc. reported that data from its first-in-human study of ONM-100, an intravenously administered imaging agent, was well tolerated and enables fluorescent visualization of tumor positive margins and occult disease in solid tumors during surgery (Press release, OncoNano Medicine, MAY 23, 2019, View Source [SID1234536557]). The full results will be presented in a poster at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place in Chicago from May 31 to June 4, 2019.

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Low pH is a well-known indicator of diseased tissue and ONM-100 has the potential to act as a broadly indicated tumor agnostic imaging agent. ONM-100 is delivered to the tumor and subsequently fluoresces in the acidic tumor microenvironment, which enables surgeons to visualize the tumor during surgery using existing near infrared surgical cameras. OncoNano recently concluded a Phase 1 clinical trial for ONM-100 at the University Medical Center Groningen (UMCG) in The Netherlands with principal investigator Professor Go van Dam. The OncoNano micelle platform is also being used to develop therapeutics, including T-cell activating agents that target lymph nodes and dendritic cells, as well as immunomodulation of the tumor micro-environment.

"pH is a simple and robust biomarker to distinguish cancer cells from healthy ones, but until now, there has not been a way to accurately visualize tumors broadly using tumor acidity in real-time during surgery and incomplete tumor resection can result in cancer recurrence and metastasis. We are thrilled with the results of our Phase 1 study which clearly demonstrated that surgeons were able to detect tumor margins and occult disease which was otherwise missed by standard of care surgery and pathology, and we look forward to expanding our trial later this year," said Ravi Srinivasan, Ph.D, founder, President and Chief Executive Officer of OncoNano.

Poster Presentation Details and Study Results:

TITLE:

Image-guided surgery for tumor agnostic detection of solid tumors using the pH-activated micellar imaging agent ONM-100

POSTER:

Poster Board: #60; Abstract 3068

DATE /TIME:

Saturday, June 1, 2019; 8:00 am – 11:00 am CT

The trial found that no ONM-100 related serious adverse events were observed and the agent was well tolerated in all 30 patients with histopathologically confirmed breast cancer, head and neck squamous cell carcinoma, colorectal cancer and esophageal cancer who were enrolled in the study. This first-in-human data demonstrate the feasibility for potential use of ONM-100 for image guided surgery, margin assessment and detection of occult disease. Tumors in four patients with breast cancer or head and neck squamous cell carcinoma that went unnoticed during surgery were detected on the margin or wound bed using ONM-100 fluorescence imaging. Additionally, two breast tumor lesions that were missed by conventional pre-operative imaging and pathological assessment were detected with ONM-100.