View this email in your browser

MedNess: bite-size biopharma and medtech news

5th May 2021
Subscribe here
MedNess This Week
HIGHLIGHTS
     Onco-I-Analyse
Janssen submits MAA for Ciltacabtagene Autoleucel in R/R MM after grant of accelerated assessment by the EMA
On 30th April, Janssen and Legend Biotech announced that the companies have filed a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for ciltacabtagene autoleucel (cilta-cel), BCMA-targeting CAR-T cell therapy, in patients with relapsed and/or refractory multiple myeloma (RRMM). 
Background: Multiple myeloma is a malignant neoplasm of plasma cells in the bone marrow. Substantial challenges and unmet needs exist for RRMM, despite therapeutics advances. Limited treatment options are available for triple-refractory patients (PI, IMiD and mAbs) with most therapies showing low, short duration responses (ORR: 20-30%) and poorer survival. CAR-Ts have emerged as promising treatment approaches for various lymphomas and leukemias. B-cell maturation antigen (BCMA) is highly expressed on MM cells and thus, is a suitable therapeutic target. ​
Details: The submission is based on the Ph 1b/2 CARTITUDE-1 study evaluating cilta-cel in 126 MM patients treated with at least 3 prior therapies including an IMiD, a PI and anti-CD38 Ab or are double refractory to an IMiD and PI. Results from the trial were presented during ASH in December 2020. In 97 efficacy evaluable patients with median of 6 prior therapies (range 3-18) (88% triple-refractory, 42% penta-refractory, 99% refractory to last therapy), ORR was 97% (sCR: 67%, VGPR: 26%, PR: 4%). At a mFU of 12.4 months, responses were rapid and durable responses with median time to first response being 30 days and ongoing responses in 72% patients. mPFS was not reached with 12-month PFS and OS rates of 77% and 89%, respectively. 93% patients (53/57) were MRD negative at 10-5.
Earlier in February, the CHMP had granted accelerated assessment to cilta-cel. In April 2019, Cilta-cel was also granted PRiME designation by the EMA.
Also in April 2021, Janssen completed the rolling submission of the BLA to the FDA, seeking approval of cilta-cel in RRMM.
Implications: Both the FDA and EMA approvals for cilta-cel could come by the end of 2021. Just last month, the FDA approved BMS’ Abecma making it the first, anti-BCMA CAR-T approval for RRMM with EMA review ongoing. However, some analysts believe that Abecma might not get the first-mover advantage given cilta-cel’s superior clinical profile. Meanwhile, BMS has been ramping up manufacturing facilities for CAR-T production.
Similar or other key competing modalities include BCMA ADCs - GSK’s Blenrep (BCMA ADC approved in August 2020), AstraZeneca’s MEDI2228, BMS’ CC-99712; anti-BCMA CAR-Ts – BMS’ bb21217, CARsgen Therapeutics’ CT053; anti-BCMA BiTEs - Amgen's AMG 701, Regeneron’s REGN5458, AbbVie/Teneobio’s TNB-383B.
 
Collated by : Shilpa Rawal, PhD
Regulatory News
FDA Grants Priority Review for New Drug Application for Mobocertinib (TAK-788) as a Treatment for EGFR Exon20 Insertion+ mNSCLC
“Patients with EGFR Exon20 insertion+ mNSCLC face considerable challenges, as current treatment options provide limited benefit, resulting in poor survival outcomes,” said Christopher Arendt, head, Oncology Therapeutic Area Unit, Takeda. “We are excited to be one step closer to offering mobocertinib as an effective oral therapy for NSCLC patients with EGFR Exon20 insertions that have received prior platinum-based chemotherapy and look forward to continuing conversations with regulatory agencies in the U.S. and around the globe.”

 
Type B meeting with the U.S. FDA to discuss Ph 2b/3 clinical trials of LiPax in patients with low-grade highly recurrent NMIBC Successful
“LIPAC Oncology is pleased to reach this important point in the development of LiPax, bringing us one step closer to providing this therapeutic option to the patients who could benefit from a novel approach to treatment,” said Will Robberts, President of LIPAC Oncology. “We have made significant progress with the program, including executing a successful Phase 1/2a trial, securing a strategic partner in South Korea and expanding the global intellectual property protection with formulation patents being granted in Europe, Japan, China, and other territories.”
Click here for more Regulatory News
Trial Results
Masitinib Ph 2B/3 study (AB12003) in mCRPC met the predefined primary endpoint of PFS improvement
  • Masitinib Ph 2B/3 study (AB12003) in mCRPC patients eligible to chemotherapy met its predefined primary endpoint.
  • Patents related to study AB12003 results are being filed and detailed results of study AB12003 will be presented soon.
  • Study AB12003 was an international, multicenter, randomized, double blind, placebo-controlled, 2-parallel group, Phase 3 study in metastatic castrate resistant prostate cancer (mCRPC) eligible to chemotherapy.
  • The study aimed to compare the efficacy and safety of masitinib (6.0 mg/kg/day) in combination with docetaxel versus placebo in combination with docetaxel. Docetaxel was combined with prednisone.
Updated results announced from Ph 1/2 pilot trial of GC4419, versus placebo, in patients with locally advanced pancreatic cancer (LAPC) who are undergoing stereotactic body radiation therapy (SBRT)
“The data from this trial continue to impress, showing consistent and durable benefits across multiple measures, including the most important for the patient, overall survival,” said Mel Sorensen, M.D., President and CEO of Galera. “These encouraging results informed the design of our Phase 2b GRECO-2 trial and underscore our excitement about the potential to make a meaningful difference for patients with this tough diagnosis.”
Click here for more Trial Results
Trial/Program Status
ARTISTRY-6 Trial of Nemvaleukin Alfa Monotherapy Initiated in Patients With Melanoma
"The initiation of the ARTISTRY-6 study represents a significant milestone in the nemvaleukin development program, building on the early signals of anti-tumor activity observed with IV nemvaleukin in melanoma," said Craig Hopkinson, M.D., Chief Medical Officer and Executive Vice President at Alkermes. "Consistent with our strategy to study nemvaleukin in difficult-to-treat cancers with clear unmet need, we look forward to further evaluating the potential clinical utility of nemvaleukin monotherapy in mucosal melanoma, a rare and aggressive form of melanoma that has very limited treatment options. We recently discussed with the FDA a potential filing pathway in mucosal melanoma, pending review of data that emerge from ARTISTRY-6."
First Patient Dosed in Ph 3 Registrational Trial Evaluating AVB-500 in Patients with Platinum Resistant Ovarian Cancer
“AVB-500 provides a novel and differentiated approach to target the GAS6/AXL pathway, which we know is commonly overactive in ovarian cancer and responsible for some of the mechanisms of resistance that lead to poor outcomes for patients who have an urgent need for more effective therapies to halt the progression of the disease,” said Katherine Fuh, M.D., Ph.D., Associate Professor, Department of Obstetrics and Gynecology, Division of Gynecologic Oncology, Center for Reproductive Health Sciences, Washington University School of Medicine, St. Louis, MO. “This Phase 3 trial will enable us to evaluate the impact of AVB-500 in improving responses to chemotherapy among women with platinum resistant tumors with the goal of extending patient survival.”
Click here for more Trial Statuses
Collated by : Richa Tewari, PhD
MedNess Business
Onco-News 
Guardant Health Announces Collaboration With Daiichi Sankyo to Develop Guardant360® CDx as a Companion Diagnostic for Enhertu® in Advanced mNSCLC
“For patients with metastatic non-small cell lung cancer, personalized medicines targeting actionable mutations, as revealed by our Guardant360 liquid biopsy test, have been life-changing,” said Helmy Eltoukhy, Guardant Health CEO. “Such medicines may extend overall survival for many patients, and we are excited to work with Daiichi Sankyo, as there remains an unmet medical need to help non-small cell lung cancer patients with HER2 mutations.”
ESSA Pharma and Bayer to Evaluate the Combination of EPI-7386 and Darolutamide in Patients with Metastatic Castration-Resistant Prostate Cancer
“We are delighted to collaborate with Bayer to explore the potential clinical role of EPI-7386 in combination with Bayer’s darolutamide in patients with metastatic castration-resistant prostate cancer, who have progressed on androgen deprivation therapy,” said Dr. David. R. Parkinson, Chief Executive Officer, ESSA Pharma Inc. “Combining our two therapies will simultaneously target both ends of the androgen receptor, and potentially allow for a more potent approach to suppressing androgen activity. We look forward to investigating the combination of these therapies and their potential role together in the treatment of prostate cancer.”
Click here for more on mergers, acquisition and business news 
Collated by : Richa Tewari, PhD
Editors' Desk
Richa Tewari, PhD
Oncology News
Esha Sehanobish, PhD
MedNess Plus
Arundithi Ananthanarayanan
MedNess Reviews
Divyaanka Iyer
BioPharma News
Shilpa Rawal, PhD
Onco I-Analyse
Debarati Banik
HealthIT
Rinki Saha
Managing Editor
Shalini Roy Choudhury
Managing Editor
Nisha Peter, PhD
Consulting Editor
Abhi Dey
Consulting Editor
Share Share
Tweet Tweet
Forward Forward
Subscribe
Disclaimer
The editors take care to share authentic information.  In case of any discrepancies please write to medness.newsletter@gmail.com
The sponsors do not have any influence on the nature or kind of the news/analysis reported in MedNess. The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of Medness. Examples of analysis performed within this article are only examples. They should not be utilized in real-world analytic products as they are based only on very limited and dated open source information. Assumptions made within the analysis are not reflective of the position of anyone volunteering or working for Medness. This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment nor investment suggestions. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
Copyright © 2019 MedNess , All rights reserved.
You are receiving this MedNess Newsletter as a subscriber on the list.

Cover Image : iStock
Images : Twitter , Unsplash.com
Content Editors: Richa Tewari , Esha Sehanobish , Rinki Saha ,  Shilpa Rawal, PhD ,  Debarati Banik  , Divyaanka Iyer , Arundithi Ananthanarayanan and Abhinav Dey 
Concept and Design: Ananda Ghosh and Nisha Peter
Our mailing address is:
MedNess
1150 First Ave. King of Prussia,, PA 19406

Want to change how you receive these emails?
You can update your preferences or unsubscribe from this list.