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MedNess: bite-size biopharma and medtech news

30th September, 2020
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HIGHLIGHTS
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Onco I-Analyse
BMS and Bluebird Bio’s bb2121 BLA accepted for Priority Review by the US FDA
On 22nd September, the US FDA accepted the BLA for idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeting CAR-T being co-developed by BMS and BlueBird Bio, for priority review in multiple myeloma (MM) patients treated with at least 3 prior therapies, including an immunomodulatory (IMiD) agent, a proteasome inhibitor (PI) and an anti-CD38 antibody (Ab). The PDUFA action date has been set for March 27, 2021.
The application is based on the pivotal Ph 2 KarMMa study evaluating ide-cel in 128 heavily pre-treated and highly refractory MM patients (median prior therapies – 6) with an IMiD, a PI and anti-CD38 Ab. After a median follow-up of 13.3 months, ide-cel demonstrated an ORR of 73% (CR/sCR: 33%) across all dose ranges, median PFS of 8.8 months and median duration of response of 10.7 months. Median estimated OS was 19.4 months. <6% patients developed grade ≥3 CRS. Clinical benefit was observed across all analysed sub-groups.
The companies had earlier submitted a BLA in March 2020, for which the FDA issued a ‘Refusal to File letter’ and demanded additional data regarding the Chemistry, Manufacturing, and Control (CMC) module of the BLA. No new clinical or preclinical was requested. Complying to FDA’s request, the companies submitted a new BLA in July 2020.
Ide-cel was previously granted breakthrough therapy designation and PRIME designation by the FDA and EMA, respectively and is also currently under accelerated assessment by the EMA. Ide-cel is also the first CAR-T therapy accepted for priority review by the FDA in MM. Regulatory submissions are also planned for geographies of the US and EU.
Other key competing assets in RRMM are GSK’s BCMA-targeting ADC (BLENREP/belantamab mafodotin; approved in August 2020),  JNJ’s BCMA CAR-T (Ciltacabtagene Autoleucel; planned US/EU submissions in 2020), JNJ’s BCMAxCD3 bispecific Ab (Teclistamab; Ph 1), Amgen’s BCMA x anti-CD3 BiTE® (AMG 701; Ph 1), Poiseda’s BCMA CAR-T (P BCMA 101; Ph 2).
Collated by :Shilpa Rawal, PhD
Drug Approvals
European Commission Approves AYVAKYT® (avapritinib) for the Treatment of Adults with Unresectable or Metastatic PDGFRA D842V Mutant GISTs
"There have historically been no treatments offering hope for patients with PDGFRA D842V mutant GIST. AYVAKYT represents the first major therapeutic breakthrough for patients with GIST harboring this mutation, defining a new standard of care," said Sebastian Bauer, M.D., medical oncologist at the West German Cancer Center in Essen, and an investigator on the NAVIGATOR trial. "The NAVIGATOR trial confirmed that almost all patients with PDGFRA D842V mutant GIST achieved tumor shrinkage and clinical responses were durable. These patients have lived longer than what is expected based on historical outcomes, and side effects have been well-tolerated in most patients. With this approval, it is more important than ever to conduct mutational testing prior to first-line treatment, so that patients with PDGFRA D842V mutant GIST may begin therapy with AYVAKYT, the only effective treatment for their tumor type."
ENHERTU® Approved in Japan for HER2+ve Metastatic Gastric Cancer Patients
“Today’s approval of ENHERTU in Japan is significant as it is the first HER2 directed therapy to demonstrate an improvement in overall survival compared to chemotherapy for previously treated patients with HER2 positive metastatic gastric cancer,” said Wataru Takasaki, PhD, Executive Officer, Head of R&D Division in Japan, Daiichi Sankyo. “We are proud of the quality and speed in which we were able to deliver this second indication to patients and physicians in Japan as it highlights our commitment to transforming science into innovative therapies for patients with cancer.”
Regulatory News
BLA Submitted to US FDA for Loncastuximab Tesirine for Treatment of R/R DLBCL
“The completion of our first BLA submission to the FDA is a significant milestone for ADC Therapeutics and takes us one step further in our evolution toward becoming a commercial-stage organization,” said Chris Martin, Chief Executive Officer of ADC Therapeutics. “We are grateful to the trial participants and investigators and to all our employees for their commitment to this clinical program, and we look forward to working with the FDA to bring Lonca to patients as quickly as possible.”
FDA Fast Track Designation Granted to DKN-01 for the Treatment of Gastric & GEJ Cancer
"We are pleased with the FDA's decision to grant Fast Track designation for the development of DKN-01 to treat patients with gastric and gastroesophageal junction cancer whose tumors express high levels of DKK1," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "The designation highlights the existing unmet medical need for new and effective treatments for this patient population. We believe that DKN-01 shows promise as a novel treatment option for biomarker-selected patients with these cancers, and this designation provides us with earlier and more frequent opportunities to interact with the FDA during the development of DKN-01."
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Trial Results
TYVYT® (Sintilimab Injection) + BYVASDA® (Bevacizumab Injection) Ph 3 ORIENT-32 Study Met Primary Endpoints in 1L Advanced HCC
The principal investigator of the ORIENT-32 study, Professor Fan Jia from Zhongshan Hospital of Fudan University, stated: "In China, HCC is the fourth most common malignancy with the second highest mortality rate. More than half of new and fatal cases of HCC in the world occur in China every year. At present, sorafenib, lenvatinib and chemotherapy are the main treatments for HCC in the first-line treatment setting in China, with very limited efficacy. About 85% of HCC patients in China have the history of HBV infection, which is a quite different feature from HCC in the European and American countries. Therefore, continued clinical research in treating HCC is of great importance in China. The ORIENT-32 study confirmed that TYVYT® (sintilimab injection), in combination with BYVASDA® (bevacizumab biosimilar injection), can prolong PFS and OS in the first-line treatment of HCC. Despite the COVID-19 pandemic, all study investigators worked to overcome many challenges to continue this trial with the goal of bringing new hope to HCC patients.”
Opdivo (nivolumab) + Chemo Demonstrated Significant OS and PFS Benefits Versus Chemo in 1L Gastric and Esophageal Cancers
“Currently, the first-line standard of care for patients with advanced or metastatic non-HER2 positive gastric or gastroesophageal junction cancer is chemotherapy. While it has been an important treatment option for these patients, chemotherapy alone is associated with a marginal survival benefit of often less than one year from the time a patient’s treatment is initiated,” said Markus Moehler, M.D., Professor of Gastrointestinal Oncology, Johannes-Gutenberg University Medical Center, Mainz. “Innovative treatments are urgently needed for patients around the world who are living with these advanced or metastatic upper gastrointestinal cancers, as there are currently no approved immunotherapy options in the first-line setting.”
KEYTRUDA® (pembrolizumab) + Chemo Reduced Risk of Death by 27% Versus Chemo as 1L Treatment for Locally Advanced or Metastatic Esophageal Cancer
“Esophageal cancer is an aggressive disease that is associated with very poor survival, and there is an urgent need for advances for newly diagnosed, previously untreated patients,” said Dr. Ken Kato, chief, department of Head and Neck Medical Oncology, National Cancer Center Hospital, Tokyo, Japan. “In KEYNOTE-590, with a 27% reduction in the risk of death, the results show KEYTRUDA has the potential to change the current treatment paradigm for the first-line treatment of patients with locally advanced and unresectable or metastatic esophageal or esophagogastric junction cancer. Results also showed a median overall survival of 12.4 months for KEYTRUDA versus 9.8 months for chemotherapy.”
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Trial/Program Status
First patient dosed in Ph 2 KEAPSAKE NSCLC trial of telaglenastat (CB-839) + pembrolizumab, carboplatin and pemetrexed in 1L non-sq NSCLC patients with KEAP1 or NRF2 mutation determined by NGS
“Therapies that inhibit glutaminase in tumors with KEAP1/NRF2 pathway activation could have a meaningful clinical impact for a substantial percentage of people with NSCLC,” said Susan Molineaux, PhD, president and chief executive officer of Calithera. “We’re proud that KEAPSAKE is among the first clinical trials investigating a potential new therapy for these patients who have a poor prognosis. Based on both the clear mechanistic rationale for telaglenastat in this indication and strong preclinical data, we’re hopeful that the study will provide valuable insights.”
Click here for more Trial Status
Collated by : Richa Tewari, PhD
Medness Reviews

Elastic Nature of RNA Nanoparticles Shows Promise For Delivery of Anticancer Drugs
Researchers from the Ohio State University have found that RNA nanoparticles have elastic properties that allow them to stretch and return to their normal shape. This trait could help them deliver anti-cancer drugs to the tumor site more effectively by taking advantage of the leaky vasculature of tumors.
To test the elastic nature of RNA nanoparticles, the team subjected them to repeated pulling with dual-beam optical tweezers and discovered that even after undergoing repeated force, the RNA particles returned to their original shape.
The team then injected the RNA nanoparticles into mice to test their ability to target tumors and study their retention in the body. They found the RNA nanoparticles displayed stronger cancer-targeting properties and accumulated less in healthy organs. The RNA nanoparticles also passed through the mouse subjects' kidneys and were excreted in their urine as little as half an hour after injection, the study found.
The researchers believe that tumor specific accumulation of the nanoparticles and their short half-life would help mitigate side effects of the chemotherapy drugs.

Children's Hospital Los Angeles launches accelerator for pediatric digital health
The Children's Hospital Los Angeles is leading a digital health accelerator that will link more than 30 hospitals across the U.S., U.K. and Australia with technology companies focused on increasing digital innovations for pediatric patients. This initiative comes in the time where there has been lag in innovation in digital health in the pediatric market.  
The KidsX Accelerator program aims to help digital health startups successfully pilot and deploy digital health solutions for pediatric patients and then grow and scale those products across pediatric healthcare.
The participating hospitals have developed a list of eight focus areas including improved remote monitoring, developing virtual rehab for kids using gamification, better pediatric mental wellness using mobile apps and creating predictive analytics for health outcomes.
The program plans to use a reverse pitch model, which allows the participating hospitals to pitch their problems or challenges to the startup community and then work with the companies that have tech solutions to meet their needs.
Entrepreneurs will work closely and collaboratively with digital health leaders at children’s hospitals to determine how their product best delivers value in the pediatric market. The 13-week program will begin in early 2021 and be completely virtual.
The companies selected for the program will be mentored by the hospitals on how to prepare for the pediatric market and will be given a place to test the innovation.
Medness Business
Onco-News

ALX Oncology to Collaborate with Merck on Ph 2 trials of ALX148 + KEYTRUDA® (pembrolizumab) in Patients with Head & Neck Cancer
“ALX148 was designed for use in combination to maximize clinical activity with a range of anti-cancer agents. We believe that blocking the CD47 myeloid checkpoint pathway bridges the innate and adaptive immune response against cancer to enhance efficacy. This collaboration builds upon the compelling combination activity observed in patients with ALX148 and KEYTRUDA,” said Jaume Pons, Ph.D., Founder, President and Chief Executive Officer of ALX Oncology. “Our goal is to transform treatment options for patients with cancer by developing ALX148 as a foundational checkpoint immunotherapy.”
IDEAYA and Pfizer Expand Clinical Trial Collaboration and Supply Agreement to Evaluate Clinical Combination of IDE196 and Crizotinib in Solid Tumors Harboring GNAQ or GNA11 Mutations
"We are excited to expand our agreement with Pfizer to evaluate the clinical combination of IDE196 and crizotinib in MUM and other solid tumors with GNAQ or GNA11 mutations," said Mick O'Quigley, Vice President, Head of Development Operations, IDEAYA Biosciences. "Through our translational research we have identified cMET expression as a potential biomarker, and we are excited to explore this rational combination between IDE196 and crizotinib clinically," said Mark Lackner, Ph.D., Senior Vice President, Head of Biology and Translational Sciences.
BioPharma and MedTech
Roche acquired Ireland based company Inflazome for $451M
On 21st September 2020, Inflazome announced that it has closed a shared purchase agreement with Roche for $451M. With this deal, Roche gains full rights to Inflazome’s pipeline of oral NLRP3 inhibitors as treatments for Parkinson’s and other diseases. Roche will continue to further develop NLRP3 inhibitors to treat different spectra of diseases.
“We are delighted to close this deal with Roche, an outstanding pharmaceutical company with a broad commitment to multiple indications. With Inflazome now part of the Roche organization, Inflazome’s pioneering molecules are well-positioned to be developed quickly and effectively so they can help patients suffering from debilitating diseases,”- Matt Cooper, Chief Executive Officer, Inflazome.
Palladio Bioscience, Inc. secures $20 M series B funding to fuel its phase 3 ALERT study for Autosomal Dominant Polycystic Kidney Disease
On 25th September 2020 Palladio Bioscience, a private clinical-stage biopharma, has raised $20 million Series B funding. The financing round was led by a new investor, Samsara BioCapital, with participation from a new investor, the Roche Venture Fund, and existing investors, Medicxi and Osage University Partners. 
Palladio’s phase 3 pipeline, lixivaptan, for Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a potent, selective vasopressin V2 receptor antagonist, which slows kidney function decline in adults with ADPKD. Palladio is currently enrolling ADPKD patients into The ALERT Study (PA-ADPKD-303), the company’s first Phase 3 clinical trial for lixivaptan.
Alex Martin, Chief Executive Officer of Palladio, commented, "We are focused on developing our lead candidate, lixivaptan, for the treatment of ADPKD, an orphan disease with a significant unmet medical need that can impact many generations of a family. This Series B financing will fund our ALERT Study, a Phase 3 trial designed to assess the safety of lixivaptan in ADPKD patients who showed liver chemistry abnormalities or other signs of liver toxicity while taking tolvaptan, the only drug currently approved in the United States for the treatment of ADPKD. We are grateful for the support of the investment community in helping us advance this potential new drug and we welcome Samsara and the Roche Venture Fund to Palladio.”
Click here for mergers , aquisitions and more business news
Collated by : Richa Tewari, PhD and Rinki Saha 
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
BioPharma News
Abhi Dey
Consulting Editor
Nisha Peter, PhD
Managing Editor
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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.
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Content Editors: Richa Tewari , Esha Sehanobish , Rinki Saha ,  Shilpa Rawal, PhD ,  Debarati Banik  , Divyaanka Iyer , Arundithi Ananthanarayanan and Abhinav Dey 
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