On August 5, 2020 Legend Biotech Corporation (NASDAQ:LEGN) reported that the China Center for Drug Evaluation, National Medical Products Administration (CDE, NMPA) has recommended Breakthrough Therapy Designation (BTD) for ciltacabtagene autoleucel (cilta-cel; LCAR-B38M CAR-T cells), an investigational B-cell maturation antigen (BCMA) targeted chimeric antigen receptor (CAR) T-cell therapy being studied for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) (Press release, Legend Biotech, AUG 5, 2020, View Source [SID1234562960]).

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The BTD for cilta-cel (LCAR-B38M CAR-T cells) is based on the ongoing Phase 2 CARTIFAN-1 study being conducted in China (MMY2002, NCT03758417, CTR20181007), the ongoing Phase 1b/2 CARTITUDE-1 study of cilta-cel (JNJ-4528) being conducted in the US (MMY2001, NCT03548207) and Japan and the Phase 1, first-in-human LEGEND-2 study conducted in China (NCT03090659). Ciltacabtagene autoleucel (cilta-cel) refers to both LCAR-B38M CAR-T cells and JNJ-4528. LCAR-B38M CAR-T cell identifies the investigational product being studied in China and JNJ-4528 identifies the investigational product being studied outside of China, both of which are representative of the same CAR-T cell therapy.

The BTD procedure is part of the recently revised Drug Registration Regulation which went into effect on July 1, 2020. The BTD process is designed to expedite the development and review of therapies that are intended for treatment of serious diseases for which there is no existing treatment and where preliminary evidence indicates advantages of the therapy over available treatment options.1 Cilta-cel is the first product that has been recommended for BTD in China.2 As per the working procedure for BTD (2020 No.82) issued by NMPA on July 8, 2020, CDE had completed the review and recommended to grant the BTD on August 4th, and BTD will be granted after 5 working days of publicity period (August 5 to 12) on the CDE website.

In December 2017, Legend Biotech entered into a worldwide collaboration and license agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to jointly develop and commercialize cilta-cel in patients with multiple myeloma. Cilta-cel is a structurally differentiated CAR-T cell therapy containing a 4-1BB co-stimulatory domain and two BCMA-targeting single domain antibodies designed to confer avidity.

"Breakthrough designation recommendation by the China CDE of NMPA represents an important regulatory milestone in the continued development of cilta-cel in multiple myeloma patients in China," said Frank Zhang, PhD, CEO of Legend Biotech. "Legend, in collaboration with Janssen, will continue to advance this investigational therapy in China and globally."

Previously, the following regulatory designations have been granted to Janssen for cilta-cel:

In July 2020, the Korea Ministry of Food and Drug Safety granted orphan drug designation.3
In June 2020, the Japan Ministry of Health, Labor and Welfare granted orphan drug designation.4
In February 2020, the European Commission granted orphan designation.5
In December 2019, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation.6
In April 2019, the European Medicines Agency granted Janssen a PRIME (PRIority MEdicines) designation.7
In February 2019, the FDA granted Janssen orphan drug designation for the treatment of multiple myeloma.8
About the Clinical Development Program

CARTIFAN-1
The Phase 2 CARTIFAN-1 confirmatory trial (MMY2002, NCT03758417, CTR20181007) is being conducted in China to further evaluate cilta-cel (LCAR-B38M CAR-T cells) in patients with RRMM who have received at least 3 prior lines of therapy and have received a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD); and documented disease progression within 12 months of starting the most recent therapy. 9

CARTITUDE-1
Cilta-cel (JNJ-4528) is currently being investigated in the Phase 1b/2 CARTITUDE-1 (MMY2001, NCT03548207) pivotal study conducted in US and Japan for the treatment of patients with multiple myeloma who have received at least 3 prior lines of therapy or are double refractory to a PI and IMiD; received a PI, an IMiD and anti-CD38 antibody; and documented disease progression within 12 months of starting the most recent therapy. 10

CARTITUDE-2
The global, multi-cohort Phase 2 CARTITUDE-2 (MMY2003, NCT04133636) study, cilta-cel (JNJ-4528) is actively recruiting patients with multiple myeloma in various clinical settings. This study is being conducted to evaluate the overall minimal residual disease (MRD) negative rate of participants who receive JNJ-4528 to further explore efficacy and safety in earlier patient populations.11

CARTITUDE-4
The global, Phase 3 CARTITUDE-4 (MMY3002, NCT04181827) study, cilta-cel (JNJ-4528) is actively recruiting patients with multiple myeloma who have received 1-3 prior lines of therapy including a PI and IMiD and are refractory to lenalidomide. The study is being conducted to evaluate the efficacy of JNJ-4528 compared to standard therapies7 including daratumumab, pomalidomide and low-dose dexamethasone (DPd) or pomalidomide, bortezomib and low-dose dexamethasone (PVd).12

LEGEND-2
LEGEND-2 (NCT03090659) is an ongoing, Phase 1, single-arm, open-label, first-in-human, study of 74 patients being conducted at four participating hospitals in China evaluating the efficacy and safety of LCAR-B38M CAR-T cells for the treatment of patients with relapsed or refractory multiple myeloma.13

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.14

Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure.15 Refractory multiple myeloma is when a patient’s disease is non-responsive or progresses within 60 days of their last therapy.16,17 Relapsed myeloma is when the disease has returned after a period of initial, partial or complete remission and does not meet the definition of being refractory.18 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.19 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.20

On August 5, 2020 AVEO Oncology (Nasdaq: AVEO) reported that it has earned a $2.8 million development milestone payment from partner Kyowa Kirin Co., Ltd. (Kyowa Kirin) (Press release, AVEO, AUG 5, 2020, View Source [SID1234562959]). The milestone relates to acceptance by the Japanese Pharmaceuticals and Medical Devices Agency of an investigational new drug (IND) application for tivozanib in a non-oncology indication being developed by Kyowa Kirin.

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"This milestone marks an important step forward for AVEO’s non-oncology pipeline," said Michael Bailey, president and chief executive officer of AVEO. "We believe tivozanib’s potential in non-oncology indications is significant, and we look forward to seeing Kyowa Kirin move this formulation into the clinic."

Under the terms of AVEO’s agreement with Kyowa Kirin, in addition to the previously-paid upfront payment of $25 million to AVEO and waiver of AVEO’s obligation to make an $18 million milestone payment upon AVEO gaining U.S. marketing approval of tivozanib for renal cell carcinoma, and now the IND development milestone, Kyowa Kirin has also agreed to pay AVEO up to an additional $388 million in potential milestone payments upon the successful achievement of certain development, regulatory, and commercial objectives in non-oncology indications of tivozanib. Kyowa Kirin will also be obligated to make tiered royalty payments on the net sales of a product for these indications, ranging from a high single-digit to low double-digit percent.

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, next-generation vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI) discovered by Kyowa Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union, the United Kingdom, Norway, New Zealand and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved activity and minimal dose modifications.1,2 AVEO’s TIVO-3 trial is supporting a regulatory submission in the U.S. seeking marketing approval of tivozanib as a treatment for relapsed or refractory RCC. Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC.4 Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal, ovarian and breast cancers. Tivozanib is also being studied by partner Kyowa Kirin in non-oncology indications.

On August 5, 2020 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported that management will present at the 2020 Wedbush PacGrow Healthcare Conference on Wednesday, August 12, 2020 at 9:45 am ET (Press release, Scholar Rock, AUG 5, 2020, View Source [SID1234562958]).

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A live webcast of the presentation may be accessed by visiting the Investors & Media section of the Scholar Rock website at View Source An archived replay of the webcast will be available on the Company’s website for approximately 90 days following the presentation.

On August 5, 2020 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer and autoimmune diseases, reported that company management will participate in three upcoming conferences (Press release, Xencor, AUG 5, 2020, View Source [SID1234562957]):

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BTIG Virtual Biotechnology Conference 2020
Date: Monday, August 10, 2020
Presentation Time: 12:00 p.m. ET / 9:00 a.m. PT
2020 Wedbush PacGrow Healthcare Virtual Conference
Date: Wednesday, August 12, 2020
Presentation Time: 2:20 p.m. ET / 11:20 a.m. PT
Canaccord Genuity 40th Annual Growth Conference
Date: Thursday, August 13, 2020
Panel Time: 1:00 p.m. ET / 10:00 a.m. PT
A live webcast of the presentations at the BTIG and Wedbush conferences will be available under "Events & Presentations" in the Investors section of the Company’s website located at www.xencor.com. Replays will be posted on the Xencor website approximately one hour after the live event and will be available for 30 days.

On August 5, 2020 TargetCancer Foundation (TCF) proudly reported the initiation of the TCF-001 TRACK (Target Rare Cancer Knowledge) Study, an innovative trial that brings advances in precision medicine to people living with rare cancers and their treating physicians, right in their own communities (Press release, TargetCancer Foundation, AUG 5, 2020, View Source [SID1234562956]). TRACK aims to provide the treatment care team with individualized treatment recommendations, informed by genomic analysis and in consultation with field-leading rare cancer clinicians and researchers. TRACK challenges the traditional paradigm of clinical trial participation by incorporating remote consent, enabling patients to access genomic information and tailored treatment recommendations without the typical requirement to travel to an academic medical center.

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TargetCancer Foundation, a patient-founded not-for-profit organization, serves as the study sponsor, and is collaborating with renowned experts in the field of rare cancer, and Foundation Medicine, Inc., a molecular information company. The research team includes Principal Investigator Razelle Kurzrock, MD (University of California San Diego), as well as co-Principal Investigators Vivek Subbiah, MD (University of Texas MD Anderson Cancer Center), James Cleary, MD, PhD (Dana-Farber Cancer Institute), and Roman Groisberg, MD (Rutgers Cancer Institute of New Jersey).

"The establishment of TRACK is a significant milestone for TargetCancer Foundation as we seek to help patients with often-overlooked rare cancers identify an effective treatment path," said Jim Palma, Executive Director, TargetCancer Foundation. "In partnership with the study’s investigators and Foundation Medicine, we will drive scientific understanding of traditionally understudied rare cancers while directly enabling patients and their physicians to make better-informed, individualized targeted therapy choices."

"With the introduction of the TRACK study, we can now offer rare cancer patients as well as oncology investigators a detailed analysis of critical genomic information that holds the promise of a targeted treatment protocol for each patient," said Dr. Kurzrock, Principal Investigator. "While unprecedented advances throughout the field continue, lack of data and insights sometimes stymie progress for treating rare cancers. With TRACK we have the opportunity to advance rare cancer research and treatment through this exciting new partnership with TargetCancer Foundation and Foundation Medicine."

About the TRACK Study Design

TRACK aims to recruit 400 people in the United States diagnosed with any rare cancer, defined as incidence of six per 100,000 people per year in the United States. Given the relevance and promise of precision medicine in cholangiocarcinoma and cancer of unknown primary, TRACK will seek to enrich study enrollment for these two subsets. Once patients are enrolled, Foundation Medicine will perform comprehensive genomic profiling (CGP) using both its FoundationOneCDx test for solid tissue samples and FoundationOneLiquid test for blood samples at baseline, as well as at multiple timepoints over a one to two year period. Both the University of California San Diego and University of Texas MD Anderson Cancer Center will serve as enrolling sites for the study. In addition, patients will have the option to enroll in the study remotely with TargetCancer Foundation, without a requirement to travel to an enrolling site, allowing full participation from geographic areas that are often not served by clinical studies. A virtual molecular tumor board (VMTB) comprised of field leading oncologists, pathologists, surgeons and other experts will recommend treatment options, directed by the genomic information of each individual patient on study.

"Foundation Medicine is dedicated to advancing the field of precision medicine through innovative collaborations," says Brian Alexander, MD, MPH, Chief Medical Officer, Foundation Medicine. "We are proud to partner with TargetCancer Foundation and Drs. Kurzrock, Subbiah, Cleary, and Groisberg to create an innovative research framework that not only provides doctors and patients with more accessible options, but also supports decision-making and learns from their journey."

The resultant treatments and participant responses will be prospectively tracked for at least one year, linking molecularly-informed treatments to specific participant outcomes. Beyond potentially informing patient treatments, the data produced through TRACK will also contribute to the field’s understanding of genomics in rare cancers, meeting a critical need where such knowledge is otherwise lacking.

TargetCancer Foundation looks forward to collaborating with companies across the pharmaceutical, biotechnology and patient-centered technology industry who share a passion for rare cancers, and is grateful to Bayer for its perspective on patients with rare cancers, and commitment as a founding supporter of the TRACK Study.

To learn more about the TRACK study and how to enroll, visit www.targetcancerfoundation.org/track.