On April 23, 2019 Gracell Biotechnologies, Co., Ltd. ("Gracell"), an immune cell gene therapy company, reported it has developed FasT CAR-T, a revolutionary platform that shortens the manufacturing time of CAR-T treatments from two weeks to one day (Press release, Gracell Biotechnologies, APR 23, 2019, View Source [SID1234539456]). FasT CAR-T also lowers manufacturing costs to a fraction of previous CAR-T therapies, while showing higher potency of CD19-directed FasT CAR-T ("CD19-F-CAR-T") in B-Cell acute lymphoblastic leukemia (B-ALL) and non-Hodgkin Lymphoma (NHL), both in vitro and in vivo.

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!

With a first-in-human clinical study of CD19-F-CAR-T ongoing, Gracell said early clinical results show the therapy is safe and significantly more potent (20-40 times) than conventionally manufactured CAR-T agents (C-CAR-T) for treating B-ALL. Gracell released the data of CD19-F-CAR-T during a presentation at the Global CAR-T Cell Therapy Development Shanghai Forum, held April 16-17, 2019.

FasT CAR-T requires only one day for manufacturing (plus 7 days for releasing tests per regulatory requirement), while C-CAR-T manufacturing takes about two weeks plus 7 days for testing. Hence, FasT CAR-T reduces vein-to-vein time by an average of 12 days, which is critical for patients with rapidly progressing disease. More importantly, CD19-F-CAR-T has demonstrated superior expansion capability, with a younger and less exhausted phenotype, according to the Company.

Dr. William Wei Cao, founder, chairman and CEO of Gracell, said, "Lengthy manufacture, high cost, relapse, and ineffectiveness in solid tumors of CAR-T products are the major challenges the CAR-T industry is facing. Gracell’s mission is to develop highly effective but low cost CAR-T cancer therapies for large unmet needs. Without support from patients, their families, and clinical scientists, we wouldn’t be able to advance the very promising FasT CAR-T technology."

Gracell has been developing a series of highly effective and low-cost CAR-T products, including Dual-CAR-T, Off-the-shelf CAR-T, and Enhanced-CAR-T products for treating solid tumors, two of which will be entering IND filing by the end of this year. Next year, the company plans to file INDs for three additional products.

Led by Dr. Cao, who previously served as co-founder and CEO of a Nasdaq-listed cell therapy company, Gracell was founded in 2017. Up to today, the company has raised nearly US$100 million combined, with Series B financing led by Temasek, joined by Lilly Asia Ventures, Kington Capital, King Star Capital and Chengdu Miaoji, and Series A led by 6 Dimensions.

On April 23, 2019 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported the launch of the new VENTANA HER2 Dual ISH DNA Probe Cocktail assay for the detection of the HER2 biomarker in breast and gastric cancer (Press release, Hoffmann-La Roche, APR 23, 2019, View Source [SID1234535348]). HER2 – human epidermal growth factor receptor 2 – is an important biomarker found in breast and gastric cancers.4 Its detection and inhibition can help to more effectively manage these aggressive cancers.

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!

The VENTANA HER2 Dual ISH DNA Probe Cocktail assay is designed to be completed within the same day, providing clinicians the ability to get results back quicker than the most common methods of confirmatory testing for HER2. Results can be read using light microscopy, eliminating the need for a specialized fluorescence microscope.

"The new VENTANA HER2 Dual ISH assay advances Roche’s commitment to personalized healthcare by delivering critical information on treatment options for breast and gastric cancer patients faster," said Michael Heuer, CEO Roche Diagnostics. "Quick results are crucial in the fight against cancer, and every additional day that a clinician and a patient must wait for test results is a day too long."

This assay is currently being launched in Europe, the Middle East and Africa, as well as Latin America and Asia Pacific. It will be submitted to the U.S. Food and Drug Administration for approval.

For more information about the assay, please visit the Roche Tissue Diagnostics breast cancer IHC/ISH portfolio page or the Anatomic Pathology site.

About the VENTANA HER2 Dual ISH DNA Probe Cocktail assay
Roche is the only provider of a fully automated brightfield Dual ISH solution for the detection of HER2 amplification. With the new VENTANA HER2 Dual ISH DNA Probe Cocktail assay, Roche provides an improved brightfield assay that is fully automated on the BenchMark IHC/ISH instruments. The assay provides clear results to pathology labs more quickly, allowing clinicians to make treatment decisions earlier.
As a global leader in breast cancer diagnostics, Roche provides a comprehensive menu of both diagnostic and predictive assays, including the VENTANA HER2/neu (4B5) Rabbit Monoclonal Primary Antibody that is indicated as an aid in the assessment of breast cancer patients for whom Herceptin treatment is considered.

About Herceptin (trastuzumab)
Herceptin is a humanised monoclonal antibody designed to target and block the function of the HER2 receptor, a protein found on the outside of many normal cells and in high quantities on the outside of cancer cells in HER2-positive cancers. Herceptin binds to a specific section of the HER2 protein, inhibiting the signals it sends that encourage tumour cell growth, while also calling on the body’s immune system to attack the cancer cells.

Since it was first approved in 1998, Herceptin has been used to treat over two million patients worldwide, diagnosed with HER2-positive breast and gastric cancers. It has also become the backbone of other innovative treatments for HER2-positive breast cancer, which have continued to improve the outcomes of patients with this otherwise aggressive disease. In addition to the standard intravenous formulation, Herceptin is available in a subcutaneous (SC) formulation which was first approved in 2013. Herceptin SC represents a significant step forward in the treatment of HER2-positive breast cancer as it offers patients a faster, more convenient and less painful way to receive treatment with Herceptin.

On April 23, 2019 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported that it will report financial results for the first quarter ending March 31, 2019 on Wednesday, May 1, 2019, before the U.S. financial markets open (Press release, Alnylam, APR 23, 2019, View Source [SID1234535346]).

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!

Management will provide an update on the Company and discuss first quarter 2019 results as well as expectations for the future via conference call on Wednesday, May 1, 2019 at 8:30 am ET. To access the call, please dial 800-289-0438 (domestic) or 323-794-2423 (international) five minutes prior to the start time and refer to conference ID 4935120. A replay of the call will be available beginning at 11:30 am ET on the day of the call. To access the replay, please dial 888-203-1112 (domestic) or 719-457-0820 (international) and refer to conference ID 4935120.

A live audio webcast of the call will be available on the Investors section of the Company’s website, www.alnylam.com. An archived webcast will be available on the Alnylam website approximately two hours after the event.

On April 23, 2019 MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the JASDAQ Market of the Tokyo Stock Exchange (Code Number:4875), reported that it has received a Notice of Allowance from the U.S. Patent and Trademark Office for a pending patent application which covers MN-166 (ibudilast) for the treatment of glioblastoma (Press release, MediciNova, APR 23, 2019, View Source;p=RssLanding&cat=news&id=2395443 [SID1234535345]).

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!

Once issued, the patent maturing from this allowed patent application is expected to expire no earlier than December 2037. The allowed claims cover a method of treating a patient diagnosed with glioblastoma or recurrent glioblastoma using MN-166 (ibudilast) as part of a combination therapy. The allowed claims cover the use of MN-166 (ibudilast) with several types of combination therapies including laquinimod in addition to radiation therapy, electric field therapy, and various therapeutic agents such as temozolomide.

Yuichi Iwaki, MD, PhD, President and Chief Executive Officer of MediciNova, Inc., commented, "We are very pleased to receive notice that this new patent will be granted. We believe it could substantially increase the potential value of MN-166 as we recently initiated enrollment in a clinical trial of MN-166 in combination with temozolomide for the treatment of recurrent glioblastoma."

About Glioblastoma

According to the American Association of Neurological Surgeons, glioblastoma (GBM) is a devastating brain cancer that typically results in death in the first 15 months after diagnosis. GBM develops from glial cells (astrocytes and oligodendrocytes) and rapidly grows and commonly spreads into nearby brain tissue. GBM is classified as Grade IV, the highest grade, in the World Health Organization (WHO) brain tumor grading system. The American Brain Tumor Association reports that GBM represents 15% of all brain tumors and 56% of all gliomas and has the highest number of cases of all malignant tumors, with approximately 12,000 new cases diagnosed each year. Despite decades of advancements in neuroimaging, neurosurgery, chemotherapy and radiation therapy, only modest improvements have been achieved and the prognosis has not improved for individuals diagnosed with GBM. Median survival is 14.6 months and two-year survival is 30%. Approximately 5% of GBM patients survive longer than 36 months.

About MN-166 (ibudilast)

MN-166 (ibudilast) is a first-in-class, orally bioavailable, small molecule macrophage migration inhibitory factor (MIF) inhibitor and phosphodiesterase (PDE) -4 and -10 inhibitor that suppresses pro-inflammatory cytokines and promotes neurotrophic factors. It attenuates activated glial cells, which play a major role in certain neurological conditions. MN-166 (ibudilast)’s anti-neuroinflammatory and neuroprotective actions have been demonstrated in preclinical and clinical studies, which provide the rationale for treatment of progressive multiple sclerosis (MS) and other neurological diseases such as amyotrophic lateral sclerosis (ALS), glioblastoma (GBM) and substance abuse/addiction. MediciNova is developing MN-166 for progressive MS and other neurological conditions such as ALS, glioblastoma, substance abuse/addiction and chemotherapy-induced neuropathy. MediciNova has a portfolio of patents which covers the use of MN-166 (ibudilast) to treat various diseases including progressive MS, ALS, and drug addiction.

On April 23, 2019 Athenex, Inc. (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported that four abstracts have been accepted for presentation at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, being held May 31 to June 4, 2019 at the McCormick Place Convention Center in Chicago, Illinois (Press release, Athenex, APR 23, 2019, View Source;p=RssLanding&cat=news&id=2395278 [SID1234535344]).

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 very pleased with the abstracts that have been accepted for presentation at ASCO (Free ASCO Whitepaper) this year," said Dr. Johnson Lau, Chairman and Chief Executive Officer of Athenex. "We look forward to sharing additional information on our lead, pivotal stage Oraxol (Oral paclitaxel and HM30181A) program as well as preliminary results from our Oratecan (Oral irinotecan and HM30181A) Phase I study and preclinical proof of concept data for our new arginine deprivation therapy platform, Pegtomarginase. We believe these will demonstrate the broad applicability of the Orascovery technology for a wide range of commonly used chemotherapy agents and the depth of our oncology-focused pipeline."

Presentation details:

Title: (Abstract TPS1116) KX-ORAX-001: An open label, randomized, multicenter, Phase III registrational study to determine the safety, tolerability, and tumor response of Oraxol (HM30181A + oral paclitaxel) and its comparability to IV paclitaxel in patients with metastatic breast cancer (MBC).
Poster Session: Breast Cancer—Metastatic
Session Date, Time, Location: Sunday June 2, 2019, 8:00 AM-11:00 AM Central Daylight Time, Hall A

Title: (Abstract 1084) Oral paclitaxel in the treatment of metastatic breast cancer (MBC) patients.
Poster Session: Breast Cancer—Metastatic
Session Date, Time, Location: Sunday June 2, 2019, 8:00 AM-11:00 AM Central Daylight Time, Hall A

Title: (Abstract 3032) A Phase 1 study of the oral administration of irinotecan in combination with the potent P-glycoprotein (P-gp) inhibitor HM30181A.
Poster Session: Developmental Therapeutics and Tumor Biology (Nonimmuno)
Session Date, Time, Location: Saturday June 1, 2019, 8:00 AM-11:00 AM Central Daylight Time, Hall A

Title: (Abstract 3090) Design, engineering, and characterization of a novel long-acting (Pegylated) single isomer human arginase for arginine depriving anti-cancer treatment.
Poster Session: Developmental Therapeutics and Tumor Biology (Nonimmuno)
Session Date, Time, Location: Saturday June 1, 2019, 8:00 AM-11:00 AM Central Daylight Time, Hall A

The abstract titles are currently available on the ASCO (Free ASCO Whitepaper) iPlanner website, with the full abstracts scheduled to be published on May 15, 2019.

Athenex has received approval of the International Nonproprietary Name (INN) "encequidar" for its novel P-gp pump inhibitor molecule, HM30181A. The Company may use the nomenclature "encequidar" in future communications.

The Orascovery platform was initially developed by Hanmi Pharmaceuticals and licensed exclusively to Athenex for all major worldwide territories except Korea, which is retained by Hanmi.