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MedNess: bite-size biopharma and medtech news

29th October, 2020
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HIGHLIGHTS
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Onco I-Analyse
CRISPR Therapeutics reported positive top-line data for its Allo CAR-T therapy, CTX110, in NHL
On 21st October, CRISPR Therapeutics announced that its off-the-shelf (allogeneic), CRISPR/Cas9 gene-edited CAR-T therapy, CTX100, demonstrated early, dose-dependent clinical benefit and response rates in Phase I CARBON study in relapsed or refractory non-Hodgkin lymphoma patients with ≥2 prior lines of therapies.
CTX110 also showed an acceptable safety profile at all but the highest evaluated doses. Its anti-tumor activity was also comparable to earlier trials of autologous CAR-T therapies.
The trial evaluated 4 different dose levels of CTX110 - 30x106 (DL1; N=3), 100x106 (DL2; N=3), 300x106 (DL3; N=4) and 600x106 (DL4; N=1) and used two lots of CTX110 manufactured from different healthy donors. Deep complete responses were observed at DL2 (33%), DL3 (50%) and DL4 (100%) in patients with DLBCL and tFL and were maintained beyond 3 months.
At DL1-3, no cases of GvHD were observed despite high HLA-mismatch between patients and donors. No DLTs, infusion-related AEs or grade ≥3 CRS and ICANS cases were reported. However, patient treated at DL4 died 52 days post infusion.
Although longer follow-up is required, the effectiveness of early data validates the CRISPR-edited allogeneic CAR-T approach and supports the rationale of treating patients with CD19+ B-cell malignancies with CTX110. CRISPR continues to enroll patients in the dose expansion phase of the study. The company also looks forward to initial data from other 2 allogeneic CAR-Ts in its pipeline - CTX120 in multiple myeloma and CTX130 in RCC and T- and B-cell lymphomas in 2021.
CTX110’s direct competitor includes ALLO-501, which demonstrated in ORR of 63% and CR of 37% in 19 efficacy evaluable patients in similar patient segment (ORR: 75% in CAR-T naïve patients). Although, allogeneic CAR-Ts offer several advantages over autologous CAR-Ts, CTX110 will also have to compete with Gilead’s Yescarta, Novartis’ Kymriah and BMS’ Liso-cel (PDUFA - November 16, 2020) for market share.
Collated by :Shilpa Rawal, PhD
Regulatory News
Fast Track Designation Granted by the FDA to Ublituximab + Umbralisib Combination for the Treatment of Adult CLL Patients
Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics stated, "We are extremely pleased to have received Fast Track designation for the ublituximab plus umbralisib regimen, or the U2 combination, to treat adult patients with CLL. The application for Fast Track was based on data from the UNITY-CLL Phase 3 study that we announced earlier this year had met its primary endpoint of progression free survival. This designation holds several important advantages to potentially expedite the development and regulatory review of U2 and underscores the significant unmet medical need that still exists for patients with CLL.” Mr. Weiss continued, “We look forward to presenting data from the UNITY-CLL Phase 3 trial later this year, which we plan to use as the basis of a U2 regulatory submission for CLL.”
NDA filing for dovitinib as a treatment for RCC postponed to 2021
Steve Carchedi, CEO of the Company, noted “Although we are disappointed with the unanticipated contract manufacturing delay for our priority dovitinib program, and the resulting setback of our planned first NDA filing for this promising cancer therapeutic, we recognize the delays are a result of the ongoing coronavirus pandemic that is affecting many facets of our industry. We remain fully committed to advancing the near-term filing of our first dovitinib NDA towards hopeful U.S. approval and to bringing this beneficial cancer therapeutic to RCC patients. Moreover, we are enthusiastic about remaining on track with our planned PMA filing for the Dovitinib DRP® companion diagnostic this year.”   
FDA clears IND application for ADI-001 for treatment of NHL
“The clearance of the IND for ADI-001 by the FDA is a significant milestone in the development of CAR γδ T cell therapies by Adicet, and marks the beginning of clinical development of a deep pipeline of “off-the-shelf” γδ T cell products,” said Chen Schor, President and Chief Executive Officer of Adicet. “We are particularly excited to advance on our goal to exploit the therapeutic potential of our first in class engineered CAR γδ T cell therapy in NHL patients. We believe that ADI-001 offers the opportunity for on demand treatment, selective tumor targeting, innate and adaptive anti-tumor immune response, and durable activity in patients. We look forward to advancing our product pipeline to address additional solid and hematologic tumors.”
Trial Results
Positive Topline Results in Second Part of Ph 3 CASSIOPEIA Study of Daratumumab in Multiple Myeloma Achieved at Pre-planned Interim Analysis
“Following the positive data from the first part of the CASSIOPEIA study, we are very pleased to see this benefit. We are appreciative of the efforts of the IFM, of HOVON and of Janssen for their work on this study,” said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.
Positive Interim Data Announced from First Cohort of Patients in Ph 1/2 Clinical Trial of GC4419 in Pancreatic Cancer
“LAPC is a devastating diagnosis with limited treatment options. By combining our investigational dismutase mimetics with SBRT, we believe we have an opportunity to improve tumor response and survival outcomes. This is the first clinical data assessing the anti-cancer activity of one of our dismutase mimetics, and we are encouraged by this analysis of the initial cohort which suggests a meaningful survival benefit in pancreatic cancer,” said Mel Sorensen, M.D., President and CEO of Galera. “We look forward to presenting the interim data from the full patient population in this pilot trial on October 27.”
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Trial/Program Status
First Patient dosed in the Ph 1/2 GRECO-1 Trial of GC4711 + SBRT for NSCLC
“SBRT holds promise to deliver more efficient, targeted and potent radiotherapy to improve cancer outcomes. This trial builds on the pilot trial in combination with SBRT in patients with locally advanced pancreatic cancer and we are eager to embark on our exploration of the potential of Galera’s GC4711 to enhance the anti-cancer efficacy of SBRT,” said Mel Sorensen, M.D., President and CEO of Galera. “We’re grateful for the support provided by the SBIR grant, which further recognizes the potential of this combination.”
Ph 1 trial of CPI-613®️ (devimistat) for Pancreatic Cancer Patients initiated in Japan
“After achieving the milestone of enrolling 500 patients in our global Phase 3 trial, we believe the natural next step is to work with Ono Pharmaceutical to bring the treatment to the Japanese market,” said Sanjeev Luther, President and Chief Executive Officer of Rafael Pharmaceuticals. “Sadly, in Japan, patients with pancreatic cancer have the lowest survival rates. We are hopeful that bringing our trial to this market will allow us to help bridge the gap in survival rates and help more people around the globe.” 
Ph 1 Japanese trial of DS-1055 initiated for Patients with relapsed/refractory advanced or metastatic solid tumors
“We are pleased to initiate clinical development to further evaluate the novel mechanism behind DS-1055, which was specifically designed to decrease the number of GARP expressing regulatory T cells and restore antitumor immune response,” said Arnaud Lesegretain, Vice President, Global Oncology Development, Alpha Portfolio, Daiichi Sankyo. “Evidence suggests that DS-1055 could serve as a new type of immune-based therapy for patients with various cancers, including those resistant or refractory to checkpoint inhibitors.”
Collated by : Richa Tewari, PhD
Medness @ HealthIT
Huawei to assist Saudi Arabia in AI-capability boosting
The National Centre for Artificial Intelligence (NCAI) of Saudi Arabia has declared a collaboration with Huawei in enhancing the national AI infrastructure. NCAI is the body responsible for executing and managing AI innovations, capabilities, and strategy execution in the Saudi kingdom. According to the most recent memorandum of understanding (MoU), Huawei will assist NCAI in training Saudi AI engineers and students, and to integrate AI within the framework of Arabic language. The MoU was unveiled at the Global AI Summit 2020 by the Saudi Data and Artificial Intelligence Authority (SDAIA). Apart from enhancing the national AI capacity, the program targets the goal to identify, explore, and cultivate the national talent pool by providing them professional training to master AI tools and technologies. This is part of the onboarding program, including the Saudi university students, researchers, and developers. The venture has a larger context in the EMR (electronic medical record) adoption in Saudi hospitals. According to Charles Yang, the president of Huawei Middle East, the ambitious long-term research and development strategy of Huawei on AI is creating unprecedented opportunities through the synergy of AI with 5G connectivity, cloud, computing, and industry applications which they are excited to bring to the Kingdom to make it a data-driven economy.
France introduces a new COVID-tracing app
A recent surge in the COVID-19 infection (+40%) in one week with 41,622 new cases has prompted the French government to unveil a newer version of a COVID-tracing app. The app is an update to the StopCovid app and has been renamed TousAntiCovid (everyone against Covid). The newer version of the app was launched as a supplement to the national plans placing 38 regions under curfew restrictions from this past weekend to combat the surge in infection cases. The new version of the app will have "information centre" with updates and statistics on local measures and will be integrated with travel certificates used for areas affected by curfews. It will also use a centralized system sending the tracing data from a phone to government systems to do the contact matching. Although seeing 500,000 downloads during the first 24 hours of introduction to the app, a stability issue was encountered initially, which was fixed overnight, according to the digital minister of France, Cedric O.
Medness Reviews

Stanford Medicine Launches COVID-19 Testing Study  of Greater San Francisco Area
In an effort to monitor clusters and build early warning systems for future outbreaks of COVID-19, Stanford University and its school of medicine have launched plans to survey the population of greater San Francisco.
The Community Alliance to Test Coronavirus at Home, or CATCH study aims to estimate the true prevalence of the disease among the population of 8.5 million people living in the Bay Area by using a combination of self-collection testing kits and online reporting.
The study is currently seeking to enroll participants, who would report their exposures and symptoms through and online portal. If selected, Home test kits developed in collaboration with the Chan Zuckerberg Biohub, which is also helping to fund the study—will be delivered through the mail within 24 hours at no cost, and will contain a gentle nasal swab for self-collection. Samples will then be processed at Stanford’s laboratories. Results of the test would be available on a private website within 48 hours after the samples are picked up. Additional tests could may be delivered if needed.
The study aims to better understand the mechanics of viral spread, symptoms and identify communities that are most vulnerable. Ultimately, the study hopes to scale up a diagnostic infrastructure that can provide fast remote testing to a broad and representative sample of the population and hopefully reduce spread in vulnerable communities.

BridgeBio Inks New Collaboration Deals with Academic Institutions
BridgeBio Pharma, which focuses on developing treatments for genetic diseases may be gearing up for its first approvals, however, it does not plan to slow down in its early-stage research.
The company recently closed a pair of R&D deals with the University of Colorado and the Salk Institute—its third and fourth academic collaborations of the year.
The deals are aimed at commercializing research out of Salk Institute and the University of Colorado’s Anschutz Medical Campus on genetically driven diseases. The company struck similar deals with the University of Florida and Johns Hopkins University this summer.
BridgeBio, which started out in 2015 to advance treatments for neglected rare diseases takes an open-ended approach to build its portfolio of assets and partnerships and collaborations with academic research institutes to accelerate promising research to clinical trials is one of the many approaches that BridgeBio has been consistently leveraging.
Medness Business
Onco-News

First patient dosed in monalizumab Ph 3 trial triggers $50M payment from AstraZeneca
“We are very pleased that our key late-stage asset, monalizumab, has progressed into Phase 3 with our partner, AstraZeneca. The launch of INTERLINK-1 represents an important financial milestone for Innate, as it triggers a $50 million milestone payment that fortifies our cash position through the end of 2022,” said Mondher Mahjoubi, Chief Executive Officer of Innate Pharma. “The steady and rapid progress of this program is a testament to our strong collaboration with AstraZeneca, and we are hopeful that together we will bring a novel treatment option to this patient population with high unmet medical need.”
Immune Therapeutics, Inc. Announces Signing of Definitive Merger Agreement Between Cytocom and Cleveland BioLabs
Michael K. Handley, President and Chief Executive Officer of Cytocom, stated, “Our merger with Cleveland BioLabs and its subsequent immune-focused platform will be a transformative growth opportunity for Cytocom and Cleveland BioLabs shareholders. We believe that the combination of these highly complementary late-stage pipelines will strengthen our position and advance our efforts to unlock the potential of immune-modulating agents in the treatment of serious medical conditions. Further, this merger will enhance our ability to become a recognized leader in immune-modulating treatments and builds on the momentum created by our recent acquisition of ImQuest Life Sciences. We plan to utilize the combined platform to further drive value with additional clinical and commercial products and continue to seek strategic partnerships and acquisitions.”
BioPharma and MedTech
Bayer acquires Asklepios BioPharmaceutical for $2 billion
On 26th October 2020, Bayer AG announced the acquisition of US-based biotech company Asklepios for $2 billion upfront and as much as a further $2 billion based on success milestones. With this giant acquisition, Bayer will gain the full rights to AskBio’s gene therapy platform, including a broad intellectual property portfolio and an established contract development and manufacturing organization (CDMO).
“In line with our purpose ‘science for a better life’, we are committed to bringing significant improvements for patients through innovation. With this acquisition, Bayer significantly advances the establishment of a cell and gene therapy platform that can be at the forefront of breakthrough science, contributing to preventing or even curing diseases caused by gene defects and further driving company growth in the future.”- Werner Baumann, CEO Bayer AG.
Ultragenyx and Solid Biosciences signed a strategic collaboration to advance the gene therapies for Duchenne Muscular Dystrophy
On 23rd October 2020, Ultragenys and Solid BIoscie announced a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne. According to the pact, Solid Bioscience will receive $40 million upfront via equity investment at a premium; up to $255 million in milestones plus royalty payments.
“We believe that Solid’s microdystrophin is best-in-class with its unique neuronal nitric oxide synthase binding domain. By using an AAV8 variant validated in prior human and other studies combined with our scalable, efficient HeLa producer cell line platform, we believe we can leverage our mutual strengths to develop a high-quality AAV-based treatment alternative for Duchenne.”  - Emil D. Kakkis, CEO and President, Ultragenyx.
“Ultragenyx has a demonstrated track record of success in developing and commercializing innovative therapies for rare diseases “We believe it is the partner of choice for exploring new gene therapy opportunities for patients with Duchenne.” - Ilan Ganot, Co-Founder, President and CEO, Solid Biosciences.
 
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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