On July 27, 2020 Checkmate Pharmaceuticals, Inc. (Checkmate), a clinical-stage biotechnology company focused on developing proprietary technology to harness the power of the immune system to combat cancer, reported that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to its product candidate, CMP-001, a differentiated Toll-like receptor 9 (TLR9) agonist, in combination with a programmed death receptor 1 (PD-1) blocking antibody (nivolumab or pembrolizumab) for two development programs, including (Press release, Checkmate Pharmaceuticals, JUL 27, 2020, View Source [SID1234562411]):

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initial treatment of patients with unresectable Stage III or Stage IV melanoma to prolong the time to disease progression; and
treatment of patients with unresectable or metastatic melanoma refractory to prior anti-PD-1 blockade to improve the overall tumor response rate.
The FDA previously granted Orphan Drug designation to CMP-001 for Stages IIb-IV melanoma.

Fast Track is a designation granted by the FDA to facilitate the development and review of a drug intended to treat a serious condition and for which available nonclinical or clinical data demonstrate the potential to address an unmet medical need. A product candidate granted Fast Track designation may be eligible for several benefits, including more frequent meetings and communications with the FDA review teams and, if relevant criteria are met, the potential for Priority Review or Rolling Review of a Biologics License Application (BLA) or a New Drug Application (NDA).

"These FDA designations for CMP-001 are testaments to the critical need for new drugs designed to treat patients with melanoma," said Barry Labinger, CEO of Checkmate. "We look forward to continued engagement with the FDA in advancing the development of CMP-001 in combination with PD-1 blockade in melanoma and head and neck squamous cell carcinoma."

About CMP-001

CMP-001 comprises a virus-like particle utilizing a CpG-A oligonucleotide. It is designed to trigger the body’s innate immune system via TLR9 and infiltrate the tumor microenvironment by the subsequent induction of both innate and adaptive anti-tumor immune responses. Checkmate believes CMP-001 is the only compound utilizing a CpG-A class TLR9 agonist in clinical development. For information on CMP-001 trials that are currently recruiting patients, please visit www.clinicaltrials.gov.

About Melanoma

Melanoma is a serious form of skin cancer that arises from a particular skin cell type called a melanocyte. Melanoma is a particularly dangerous form of cancer because of its ability to spread to other organs rapidly if not surgically removed at an early stage, as well as low response rates and limited durability of response when treated with commonly used chemotherapeutics. In 2020, melanoma of the skin is estimated to be the fifth most diagnosed cancer, and accounts for approximately 1% of all skin cancers in the U.S. According to the American Cancer Society, there will be an estimated 100,350 new diagnoses and approximately 6,850 patients will die as a result of melanoma in the United States in 2020 alone.

On July 27, 2020 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported that it will release its financial results for the second quarter ended June 30, 2020, after the close of market on Monday, August 10, 2020 (Press release, Castle Biosciences, JUL 27, 2020, View Source [SID1234562410]).

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Company management will host a conference call and webcast to discuss its financial results and provide a corporate update at 4:30 p.m. Eastern time on the same day.

Conference Call and Webcast Details

A live webcast of the conference call can be accessed at View Source or via the webcast link on the Investor Relations page of the Company’s website (www.castlebiosciences.com). Please access the webcast at least 10 minutes before the conference call start time. An archive of the webcast will be available on the Company’s website until August 31, 2020.

To access the live conference call via phone, please dial 877-282-2581 from the United States and Canada, or +1 470-495-9479 internationally, at least 10 minutes prior to the start of the call, using the conference ID 7388802.

There will be a brief Question & Answer session following the corporate update.

On July 27, 2020 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company, reported that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has granted priority review status to the New Drug Application (NDA) of pamiparib, BeiGene’s investigational inhibitor of PARP1 and PARP2, for the treatment of patients with deleterious or suspected deleterious germline BRCA-mutated advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy (Press release, BeiGene, JUL 27, 2020, View Source [SID1234562409]).

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"Pamiparib is our third internally developed drug candidate that has been granted priority review in China, following tislelizumab and BRUKINSA. It received priority review within a week of the acceptance of the application, reflecting the unmet need for patients with this advanced disease," commented Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene. "We look forward to presenting the clinical data that support this NDA at an upcoming medical conference and the next milestones in the pamiparib program."

The NDA of pamiparib as a potential treatment for patients with advanced ovarian, fallopian tube, or primary peritoneal cancer was accepted in July 2020. It is supported by clinical results from the pivotal Phase 2 portion of the Phase 1/2 trial (NCT03333915), which enrolled 113 patients in China with high-grade epithelial ovarian cancer (including fallopian tube or primary peritoneal cancer) or high-grade endometrioid epithelial cancer, harboring germline BRCA1/2 mutation, following at least two prior lines of standard chemotherapy. BeiGene is developing pamiparib as a monotherapy and in combination with other therapies for the treatment of a broad array of solid tumors.

Priority review and approval was established in China to facilitate drug registration management and accelerate the development of innovative drugs with significant clinical advantages and for which there is an urgent clinical need. According to the Drug Registration Regulation (Bureau Order 27) implemented on July 1, 2020, the regulatory authority will prioritize the review process and evaluation resources for applications under priority review. These applications are expected to have reduced review and approval timelines.

About Ovarian Cancer

In China, ovarian cancer is the tenth most common form of cancer among women, with over 50,000 new cases and more than 30,000 deaths in 2018.1 More than 60 percent of patients are diagnosed with advanced disease.2 The standard therapy for ovarian cancer consists of surgery followed by postoperative platinum-based chemotherapy. An estimated 70 percent of patients with epithelial ovarian cancer, which accounts for more than 90 percent of all ovarian cancer,3 who achieve a full remission following first-line therapy will develop recurrent disease.4

About Pamiparib

Pamiparib (BGB-290) is an investigational inhibitor of PARP1 and PARP2 which has demonstrated pharmacological properties such as brain penetration and PARP-DNA complex trapping in preclinical models. Discovered by BeiGene scientists, pamiparib is currently in global clinical development as a monotherapy or in combination with other agents for a variety of solid tumor malignancies. To date, more than 1,200 patients have been enrolled in clinical trials of pamiparib.

A New Drug Applications (NDA) for pamiparib for patients with ovarian cancer has been accepted and granted priority review by the Center for Drug Evaluation (CDE) of the NMPA.

About the Pamiparib Clinical Program

Clinical trials of pamiparib include:

Phase 3 trial in China of pamiparib as maintenance versus placebo in patients with platinum-sensitive recurrent ovarian cancer (NCT03519230);

Phase 2 trial of pamiparib in patients with metastatic castration-resistant prostate cancer with homologous recombination deficiency (NCT03712930);

Phase 2 trial in China of pamiparib in patients with metastatic HER2-negative breast cancer with BRCA mutation (NCT03575065);

Phase 2 trial of pamiparib in patients with advanced or inoperable gastric cancer (NCT03427814);

Phase 1/2 trial in China of pamiparib in patients with advanced ovarian cancer, fallopian cancer, and primary peritoneal cancer or advanced triple negative breast cancer (NCT03333915);

Phase 1b/2 trial of pamiparib in combination with radiation therapy and/or temozolomide in patients with first-line or recurrent/refractory glioblastoma (NCT03150862);

Phase 1b trial of pamiparib in combination with temozolomide in patients with locally advanced or metastatic solid tumors (NCT03150810); and

Phase 1b trial of pamiparib in combination with tislelizumab for a variety of solid tumor malignancies (NCT02660034).

On July 27, 2020 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that its partner, Austrianova, has submitted a Drug Master File (DMF) to the U.S. Food and Drug Administration (FDA) (Press release, PharmaCyte Biotech, JUL 27, 2020, View Source [SID1234562408]). The DMF provides all confidential and detailed information covering the production of the CypCaps final product, which was produced by Austrianova and will be used in PharmaCyte’s planned clinical trial in locally advanced, inoperable pancreatic cancer (LAPC).

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said, "We’ve reached an exciting milestone at PharmaCyte that has us on the precipice of reaching our long-awaited goal of submitting an Investigational New Drug Application (IND). We’re elated to announce that our partner, Austrianova, submitted a DMF with the FDA for the production of our Cell-in-a-Box encapsulated cell product CypCaps. This DMF is an important and significant event since it is the last prerequisite for the formal FDA application process. It will support and now facilitate the submission of our IND."

Austrianova’s Chief Technical Officer, Walter H. Gunzburg stated, "The DMF filing is a key event for both PharmaCyte and Austrianova since it will provide the basis for many Cell-in-a-Box products in addition to CypCaps. The DMF is by its nature a comprehensive document compiled from many months of in-house work and past historical data and was compiled with the support and advice of an external consultant."

A DMF is submitted to the FDA to provide detailed information about facilities, processes and materials used in the manufacturing, processing and packaging of human drugs and biologics. It is a prerequisite to securing approval and commercialization and ensures confidentiality of proprietary information related to the Active Pharmaceutical Ingredient (API). The information contained in a DMF is used to support, among other applications, an IND.

The DMF requirements are complex and specific, encompassing every detail involved with the manufacture of the API – from raw materials to analytical methods, process development and optimization. The scrutiny goes all the way back to the starting materials used in the API.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced, inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

On July 27, 2020 Celsion Corporation (NASDAQ: CLSN), an oncology focused drug-development company, reported the randomization of the first two patients in the Phase II portion of the Phase I/II OVATION 2 Study with GEN-1 in advanced ovarian cancer (Press release, Celsion, JUL 27, 2020, View Source [SID1234562407]). The Company anticipates completing enrollment of up to 118 patients in the third quarter of 2021. Because this is an open-label study, the Company intends to provide clinical updates throughout the course of treatment including response rates and surgical resection scores.

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GEN-1 was designed using TheraPlas, Celsion’s proprietary, synthetic, non-viral nanoparticle delivery system platform. It is an interleukin-12 (IL-12) DNA plasmid vector associated with a non-viral nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein.

The OVATION 2 Study combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).

"During the first half of 2020, we reported data from the Phase I portion of the OVATION 2 Study that showed successful tumor resections, with seven out of eight patients (87.5%) in the GEN-1 treatment arm having a complete tumor resection (R0), which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed. The NACT-only treatment arm had an R0 resection rate of 50%," said Dr. Nicolas Borys, Celsion’s Chief Medical Officer. "The Company engaged Medidata/Acorn to independently evaluate our data using a propensity matched synthetic control arm (SCA) with results from the Company’s previously completed Phase I studies. The results suggested that GEN-1 nearly doubled progression-free survival (PFS) in these patients. These findings are not statistically significant, however, due to the small number of patients, but nonetheless are encouraging and supportive of our current Phase II study."

"This past May we reported that the Data Safety Monitoring Board recommended that the Phase II portion of the OVATION 2 Study proceed with a GEN-1 dose of 100 mg/m²," said Michael H. Tardugno, Chairman, President and Chief Executive Officer of Celsion. "Patients will undergo up to 6 months of immunostimulatory GEN-1 treatment. Based on results from earlier studies, we believe this regimen holds great potential to alter the current treatment paradigm, and in doing so improve survival for ovarian cancer patients, whose prognosis is generally poor. Our investigators are among the leading researchers in ovarian cancer. Together, we are delighted to have begun enrolling patients in the Study and look to achieve our goal of providing new novel therapeutic options to patients with this difficult-to-treat cancer."

In March 2020 the Company announced the European Medicines Agency Committee for Orphan Medicinal Products recommended that GEN-1 be designated as an orphan medicinal product for the treatment of ovarian cancer. GEN-1 previously received orphan designation from the U.S. Food and Drug Administration for the treatment of ovarian cancer. This designation by the EMA provides 10 years of exclusivity once approved for marketing.

About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer. and recently completed a Phase Ib trial of GEN-1 in combination with PEGylated doxorubicin in patients with platinum-resistant ovarian cancer.