Heavy! The legendary BCMA CAR-T was approved for listing by the Ministry of Health, Labor and Welfare of Japan

September 28, 2022 / Medical MedClub News / - On September 27, 2022, local time, Legendary Creatures (NASDAQ: LEGN) officially announced that the Ministry of Health, Labor and Welfare (MHLW) of Japan has approved CARVYKTI^® (Ciltacabtagene Autoleucel, abbreviated as Cilta-cel) is used to treat adult patients with recurrent or refractory multiple myeloma (R/R MM) only if the following two conditions are met:

• Patients have no history of chimeric antigen receptor (CAR)-positive T cell infusion therapy targeting B-cell mature antigen (BCMA);
  
• Patients have previously received at least three lines of therapy, including proteasome inhibitors (PI), immunomodulators (IMiD), and anti-CD38 monoclonal antibodies, and have not responded to the last treatment or have relapsed
  .
  

The application for the new drug launch was submitted
by legendary biopartner Janssen.
In December 2017, Legendary Biologics signed an exclusive global licensing and cooperation agreement with Janssen Corporation to develop and commercialize Cilta-cel
.

▲ Image source: The legendary biological
CARVYKTI^® features
two single-domain antibodies to the targeted B cell mature antigen (BCMA), personalized customization, and one-time infusion of the drug.
The approval is based on the results of the critical Phase 1b/2 CARTITUDE-1 study, which included 97 patients
who had previously received median six-line therapy (range 3-18), including PI, IMiD, and anti-CD38 monoclonal antibodies.
Studies have shown that non-Japanese people receiving one-time treatment at Cidaki Ollense showed a deep and lasting response rate of 96.
9% (95% CI, 91.
2-99.
4 n=97)
in the overall response rate (ORR).
Notably, 65 patients achieved a complete response in the strict sense (sCR), with a complete response rate of 67% in the strict sense (95% CI, 56.
7-76.
2), i.
e.
, no signs or symptoms of the disease can be observed by imaging or other tests after treatment*
.
The efficacy observed in Japanese patients with multiple myeloma is consistent with that of non-Japanese populations+
.
At a median follow-up of 18 months, the median duration of response (DOR) was 21.
8 months#
.

The CARTITUDE-1 study evaluated the safety of Sidakhiolaense in 106 adult patients, including 97 non-Japanese people and 9 Japanese patients
.
Of the 106 patients treated with Sidaki Olense, 105 (99.
1%) developed adverse reactions
.
The most common adverse effects included cytokine release syndrome (94.
3%), cytopenia (79.
2%), neutropenia (75.
5%), thrombocytopenia (59.
4%), anemia (51.
9%), neurological adverse events (39.
6%), infection (19.
8%), and hypopropiglobulinemia (11.
3%)
.

*Data as of September 1, 2020, i.
e.
at least 6 months
after treatment of the last patient.

+Data as of 22 July 2021, i.
e.
the last patient was treated for at least 12 months
.

# The data was presented
at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.

In February this year, CARVYKTI^® (Siddaki Olensai) was approved for marketing by the US Food and Drug Administration (FDA) and in May it received conditional marketing authorization
from the European Commission (EC).

"The approval of CARVYKTI^® for marketing by Japanese regulators is another important milestone
in our journey to help patients with recurrent or refractory multiple myeloma worldwide.
The cooperation with Janssen not only lays a solid foundation for the drug to enter the global market, but also helps the clinical development program continue to move forward
.
We strongly believe that Cilta-CEL will provide a vital treatment option
for patients with multiple myeloma.
”

Dr.
Ying Huang

CEO of Legendary Creatures

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About CARVYKTI^® (Ciltacabtagene Autoleucel, abbreviated as Cilta-cel)

CARVYKTI^® is a chimeric antigen receptor T cell (CAR-T) therapy that targets B-cell mature antigen (BCMA), using transgenes from chimeric antigen receptor (CAR) to modify a patient's own T cells to identify and eliminate BCMA-expressing cells
.
BCMA is mainly expressed on the surface
of malignant multiple myeloma B cells, advanced B cells and plasma cells.
CARVYKTI's^® CAR protein has two BCMA-targeted single-domain antibodies that have a high affinity for BCMA-expressing cells, and after binding to BCMA-expressing cells, CAR can promote T cell activation, expansion, and subsequent clearance of target cells
.

In December 2017, Janssen signed an exclusive global licensing and cooperation agreement with Legendary Biologics to develop and commercialize Cilta-cel
.

CARVYKTI^® was approved by the US FDA in February 2022 for the treatment of adult patients with
recurrent or refractory multiple myeloma.
In May 2022, the European Union EC granted CARVYKTI^® a conditional marketing authorization
.
Cilta-CEL received breakthrough therapy designation
in the United States in December 2019 and in China in August 2020.
In addition, Cilta-CEL was awarded
the European Commission's Preferred Drug Qualification in April 2019.
The U.
S.
FDA, EUEA, and Japan PMDA granted Cilta-cel orphan drug designation
in February 2019, February 2020, and June 2020, respectively.
In May 2022, the Orphan Medicines Committee of the European Medicines Agency unanimously recommended that the orphan drug designation
of Sidakhiolensee be maintained based on clinical data (with improved and persistent rates of complete response after treatment).

About CARTITUDE-1

CARTITUDE-1 is a 1b/2, open-label, monobrachial, multicenter study to assess the safety and efficacy of Cilta-cel in the treatment of adults with relapsed or refractory multiple myeloma who have received at least 3-line pre-treatment, including proteasome inhibitors (PI), immunomodulators (IMiD), and anti-CD38 monoclonal antibodies, and disease progression
on the last treatment.
All patients in the study had previously received median sixth-line therapy (range 3-18
).
Of the 97 patients who participated in the study, 99 percent were intolerant of the last line of treatment, and 88 percent were refractory to three types of treatment, meaning they did not respond to IMiD, PI, and anti-CD38 monoclonal antibodies, or no longer responded
.

About multiple myeloma

Multiple myeloma is an incurable blood cancer that originates in the bone marrow and is characterized
by excessive proliferation of plasma cells.
According to statistics, in Japan, about 7800 people were diagnosed with multiple myeloma in 2018, and about 4200 patients died
in 2020.
Although some patients with multiple myeloma have no obvious symptoms, most patients are diagnosed
with symptoms such as bone problems, low blood counts, elevated calcium, kidney problems, or infections.
Although there may be some relief after treatment, unfortunately, patients are likely to relapse
.
Patients who relapse after treatment with standard therapies, including protease inhibitors, immunomodulators, and anti-CD38 monoclonal antibodies, face poor prognosis and limited treatment
.

About the legendary creatures

Founded in 2014, Legendary Biologics (NASDAQ: LEGN) is a multinational biopharmaceutical company integrating R&D, clinical, production and commercial development of tumor immune cell therapy, ranking first in the global field of immune cell therapy, with a total of more than 1,000 employees worldwide
.
At present, Janssen and Janssen have successfully pushed the core product CARVYKTI to the US FDA for approval and listing, and obtained the EU conditional marketing authorization, which is expected to solve the world-class problem
of multiple myeloma treatment.
In addition, the company has a number of in-research cell therapies for the treatment
of hematoma, solid tumors and other difficult diseases.

The company has been selected as the special fund project for the development of strategic emerging industries in Jiangsu Province, "Jiangsu Precision Cell Immunotherapy Engineering Technology Research Center", "Jiangsu Graduate Workstation", "Jiangsu Invisible Little Giant Enterprise", "Nanjing Engineering Research Center", "Nanjing High-end R&D Institution", "Nanjing Postdoctoral Innovation Practice Base", "Nanjing Unicorn Enterprise", "China Unicorn Enterprise", and won the "Jiangsu Provincial Science and Technology Progress Award", "Kunpeng Award for Innovation in China's Biomedical Industry Chain", " China Top 100 Pharmaceutical Innovation Enterprises", "Nanjing Talent Introduction and Talent Demonstration Enterprise Golden Wutong Award", "Massachusetts Science and Technology Review" 50 Smart Companies", "Top 20 Most Influential Innovative Therapy Enterprises" and other honors
.
For more information, please visit: www.
legendbiotech.
cn