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MedNess: bite-size biopharma and medtech news

26th May, 2021
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MedNess This Week
HIGHLIGHTS
Onco-I-Analyse
Janssen’s Rybrevant (amivantamb) receives FDA accelerated approval for NSCLC patients with EGFR ex20 insertion mutations
On 21st May, the FDA granted accelerated approval to Janssen’s Rybrevant (amivantamb; EGFR-MET bispecific antibody) for locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations and disease progression on or after platinum-containing chemotherapy. The FDA simultaneously approved the Guardant360 companion diagnostic for detection of EGFR ex20 mutations in patients likely to benefit from Rybrevant.
Background: Nearly 2-3% NSLC tumors have EGFR exon 20 insertion mutations, which are the 3rd most prevalent activating EGFR mutations. Patients with these mutations have worse prognosis and are generally insensitive to treatment with TKIs.
Details: The approval was granted based on ORR and DOR data from the Phase 1 CHRYSALIS study which is evaluating amivantamab monotherapy and in combination with lazertinib in various sub-populations of NSCLC patients, including those with EGFR exon 20 insertion mutations and platinum-treated disease. 46% and 25% patients had received prior immunotherapies and TKI, respectively with a median of 2 prior therapies. In 81 efficacy evaluable patients, amivantamab monotherapy demonstrated an ORR (BICR) of 40% (CR: 3.7%, PR: 36%) and mDOR of 11.1 months, with 63% patients having DOR of ≥6 months. Patients lived for a median of 22.8 months and had median PFS of 8.3 months.
Rybrevant was granted breakthrough therapy designation in March 2020, and priority review in December 2020.
The application was evaluated under the FDA’s Project Orbis, in collaboration with international regulatory bodies (Brazilian Health Regulatory Agency and UK’s MHRA) and used Assessment Aid leading to approval 2 months prior to its PDUFA date. Reviews are underway with European and other international agencies.
The current approval is contingent upon the continued clinical benefit in confirmatory trials. Janssen is evaluating amivantamab+chemotherapy in a Phase III PAPILLON trial for NSCLC patients with EGFR ex20 insertion mutations.
Janssen’s CarePath is an amivantamab access and support program for both HCPs and patients.
Implications: Rybrevant has carved a niche segment being the first targeted and bispecific antibody therapy for this subpopulation of lung cancer. Takeda’s mobocertinib (oral TKI) is also under priority review for the same patient segment. Janssen is also evaluating amivantamab in broader populations to expand its utilization.
Collated by : Shilpa Rawal, PhD
Drug Approvals
FDA Approves Opdivo® as Adjuvant Treatment of Completely Resected Esophageal or GEJ Cancer in Patients who have Received Neoadjuvant Chemoradiotherapy
“Locally advanced esophageal and gastroesophageal junction cancers are aggressive tumor types that often require multiple approaches to address the disease, including chemotherapy, radiation and surgery,” said Ronan J. Kelly M.D., MBA., director, Baylor Scott & White Charles A. Sammons Cancer Center, and W.W. Caruth Jr. Endowed Chair of Immunology at Baylor University Medical Center. “Even after neoadjuvant CRT followed by surgery, there may be a high risk of recurrence for patients who do not achieve a pathologic complete response. In the CheckMate -577 trial, we saw a doubling in median disease-free survival compared to placebo, which suggests that Opdivo could become a new standard of care for these patients.1,9 This is exciting news, providing renewed hope.”
Regulatory News

Breakthrough Therapy Designation for VS-6766 with Defactinib in Recurrent Low-Grade Serous Ovarian Cancer
“Patients with low-grade serous ovarian cancer urgently need better solutions due to low response rates and tolerability issues associated with current therapies,” said Melissa Aucoin, CEO of the National Ovarian Cancer Coalition. “A Breakthrough Therapy designation in this disease is a significant step forward for the women who often, at a relatively young age, start a lengthy battle with this highly recurrent and impactful disease.”
US FDA approves IND application for CpG-STAT3 siRNA therapy
Dr Kortylewski: “Our laboratories were the first to demonstrate that successful cancer immunotherapy needs to be two-step since TLR9 immunostimulation is only effective when STAT3 in the tumour microenvironment is no longer active,” Kortylewski added. “It is exciting to see this technology approaching clinical application with a strong ally in biopharma.”
Click here for more Regulatory News
Trial Results
Positive Topline Results Announced from Ph 3 Trial of Tislelizumab + Chemotherapy Combination as First-Line Treatment for Recurrent or Metastatic Nasopharyngeal Cancer
“We are excited to see a clinically meaningful improvement in progression-free survival in our Phase 3 trial for tislelizumab plus chemotherapy in patients with NPC. This is our fifth positive Phase 3 readout for tislelizumab, which we are developing broadly as a potentially differentiated anti-PD-1 antibody,” said Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology, at BeiGene. “We are grateful for the patients and clinicians who participated in this trial and hopeful that they may have a new treatment option in the future.”
"For oncologists and people living with metastatic breast cancer, these data provide insights into HALAVEN real-world practice," said Dr. Takashi Owa, Vice President, Chief Medicine Creation Officer and Chief Discovery Officer, Oncology Business Group at Eisai. "We have remained committed to the continued data generation for HALAVEN, both in the real-world setting and in translational research related to mBC, to drive our continued innovation for difficult-to-treat diseases like mBC."
Trial/Program Status
First Patient Dosed in Ph 1 Trial of MB-101 (IL13Rα2-specific CAR T cells) to Treat Leptomeningeal Brain Tumors
Lisa Feldman, M.D., Ph.D., a neurosurgeon and assistant clinical professor in the Division of Neurosurgery at City of Hope and principal investigator of the clinical trial, commented, “Based on our research to date, including a previous clinical trial at City of Hope, further evaluation is warranted for this CAR T cell therapy. The prior clinical trial demonstrated encouraging potential of administering autologous IL13Rα2-CAR T cells intraventricularly to help treat patients with leptomeningeal brain tumors, a form of metastatic brain cancer that is difficult to treat. We continue to work closely with the Mustang team to potentially bring a safe, effective treatment option to patients suffering with this life-threatening disease.”
FDA Approves IDE Supplement for Ph 3 Pivotal LUNAR Trial of Tumor Treating Fields in NSCLC
“We are very pleased with the FDA approval of the DMC’s recommended protocol adjustments and are grateful for the FDA’s prompt review,” said Asaf Danziger, Novocure’s CEO. “We now anticipate last patient enrollment in the LUNAR trial in the fourth quarter of 2021 with final data available in 2022 and look forward to sharing data from the LUNAR trial as quickly as possible.”
Click here for more Trial Statuses
Conference Coverage: Highlights from American Society of Clinical Oncology (ASCO) 2021
  1. AbbVie to Showcase Breadth of Oncology Portfolio and Pipeline
  2. AB Science announced results from masitinib study AB12005 in pancreatic cancer
  3. ADC Therapeutics Announces Posters to highlight ZYNLONTA™ LOTIS-2 subgroup analyses, ZYNLONTA™ LOTIS-5 confirmatory trial and Cami in advanced solid tumors
  4. Allogene Therapeutics CD19 Forum Highlights Positive Results from Ph 1 Studies of ALLO-501 and ALLO-501A in R/R NHL and Plan to Initiate Pivotal Study in 2021
  5. Amgen To Showcase New Data From Oncology Portfolio
  6. Astellas to Present Data from Expanding Oncology Portfolio
  7. AstraZeneca data support ambition to revolutionise cancer outcomes by treating earlier and transforming the patient experience
  8. AVEO Oncology Announces Positive Results from Randomized Ph 2 Study of Ficlatuzumab in Combination with Cetuximab in Pan-Refractory, Metastatic HNSCC
  9. AVEO Oncology Announces Presentation of Long-Term Efficacy Follow Up, Additional Tolerability Data from the Ph 3 TIVO-3 Study of FOTIVDA® (tivozanib) in R/R RCC
  10. BeiGene to Showcase Broad Clinical Portfolio
  11. BMS Announces Relatlimab and Nivolumab Fixed-Dose Combination Significantly Improves PFS vs. Opdivo in Patients with Previously Untreated Metastatic or Unresectable Melanoma
  12. Clovis Oncology Highlights Rubraca® (Rucaparib) And Lucitanib Data
  13. Daiichi Sankyo Advances Science Across Three Lead DXd ADCs with New Data in Multiple Cancers
  14. Data from EMD Serono’s Oncology Portfolio Highlight Significant Advances in Cancer Care
  15. Early amcenestrant data support its potential to become a new endocrine backbone therapy for ER+/HER2- breast cancer
  16. Eisai to present data on oncology pipeline and products
  17. Four-Year Data from Phase 3 CheckMate -227 Trial Show Durable, Long-Term Survival with Opdivo + Yervoy (ipilimumab) in NSCLC Patients with PD-L1 Expression ≥1%
  18. Genmab Announces Abstracts Evaluating Products in Pipeline, Portfolio
  19. Gilead-Kite Oncology to Present Transformative Science From Growing Portfolio
  20. IMBRUVICA® (ibrutinib)-Based Combination Regimen as a Fixed-Duration, 1L Treatment for CLL Demonstrates High Rates of Disease Control
  21. ImmunoGen Announces Mature Data from FORWARD II Study Evaluating Mirvetuximab Soravtansine in Combination with Avastin® in Recurrent Ovarian Cancer, Regardless of Platinum Status
  22. Iovance Biotherapeutics Announces Clinical Data Updates for Lifileucel in Advanced Melanoma
  23. Janssen Presents Updated Data on First-in-Class Talquetamab Suggesting Deep and Durable Responses in Heavily Pretreated Patients with Multiple Myeloma
  24. Janssen’s Updated Ph 1 Results for Teclistamab Suggest Deep, Durable Responses in Patients with Heavily Pretreated Multiple Myeloma
  25. Karyopharm Announces XPOVIO® (selinexor) Data
  26. LIBTAYO® (CEMIPLIMAB-RWLC) presentations highlight expanding clinical data in diverse cancers
  27. Lilly Announces Details of Presentations
  28. Long-Term Data from Pivotal KarMMa Study Continue to Demonstrate Deep and Durable Responses and Predictable Safety Profile with BMS and bluebird bio’s Abecma (idecabtagene vicleucel) in RRMM
  29. Long-term Data on Vitrakvi® (larotrectinib) Further Demonstrate Strong Clinical Profile in Patients with TRK Fusion Cancer Regardless of Tumor Type and Age
  30. MEI Pharma and Kyowa Kirin to Present Clinical Data from Ph 1b Study of Zandelisib in Combination with Zanubrutinib
  31. Merus Announces Publication of Abstract on Zenocutuzumab in NRG1-fusion (NRG1+) Cancers
  32. Novartis to unveil new data from its robust portfolio, including overall survival in prostate and breast cancer
  33. Opdivo + Yervoy with Two Cycles of Chemotherapy Demonstrates Durable Overall Survival vs. Chemotherapy at Two Years in 1L NSCLC in Phase 3 CheckMate -9LA Trial
  34. Pivotal Phase III data at ASCO show Tecentriq helps certain people with early lung cancer live significantly longer without their disease returning
  35. Precision BioSciences to Present Data from its First Generation CD19 Allogeneic CAR T Program in Relapsed/Refractory Non-Hodgkin Lymphoma
  36. Sanofi to showcase data from its transformative oncology pipeline
  37. Seagen and Astellas Announce Updated Results from Two Trials of PADCEV® (enfortumab vedotin-ejfv) in Patients with Locally Advanced or Metastatic Urothelial Cancer Not Eligible for Cisplatin Chemotherapy
  38. Six-and-a-Half-Year Outcomes for Opdivo + Yervoy Continue to Demonstrate Durable Long-Term Survival Benefits in Patients with Advanced Melanoma
  39. Takeda Presents Updated Results for Mobocertinib, Further Substantiating the Clinical Benefit in Patients with EGFR Exon20 Insertion+ mNSCLC
  40. Takeda to Present Positive Primary Analysis from Ph 2 OPTIC Trial of ICLUSIG® (ponatinib), Reinforcing Ability to Address Gaps in Care for Chronic-Phase CML
  41. Updated Amivantamab and Lazertinib Combination Data Demonstrate Durable Responses and Clinical Activity for Osimertinib-Relapsed Patients with EGFR-Mutated NSCLC
  42. Veru Announces Acceptance of Two Abstracts for Presentation
Collated by : Richa Tewari, PhD 
       MedNess @ HealthIT
Google launches AI-powered tool for dermatology-related issues
Looking at the volume of searches every year on hair/skin/nail related queries, Google Health have come up with a web-based tool (to be unveiled later this year) where related dermatological queries can be answered through inspection by AI. The process would be as simple as uploading pictures of the issue from different angle, answering a few questions on user’s skin type, how long they’ve had the issue, and other symptoms., and getting recommendations based on a database of 288 conditions, for the user then to search further.  Google debuted the web-based application at its annual I/O developer conference on May 18.
According to a blog post written by Peggy Bui, MD, product manager at Google Health, and Yuan Liu, PhD, technical lead at Google Health, “The tool is not intended to provide a diagnosis nor be a substitute for medical advice as many conditions require clinician review, in-person examination, or additional testing like a biopsy. Rather we hope it gives you access to authoritative information so you can make a more informed decision about your next step.” The team has published the details of the tool in a 2020 Nature Medicine publication. In summary, it is a ‘deep learning system (DLS) to provide a differential diagnosis of skin conditions using 16,114 de-identified cases (photographs and clinical data) from a teledermatology practice serving 17 sites. Using 963 validation cases, the study utilized a rotating panel of three board-certified dermatologists who defined the reference standard. The DLS was non-inferior to six other dermatologists and superior to six primary care physicians (PCPs) and six nurse practitioners (NPs) (top-1 accuracy: 0.66 DLS, 0.63 dermatologists, 0.44 PCPs and 0.40 NPs). 
Pediatric blood clots modeled through predictive learning tool
In a study conducted by the Pediatric Hematology-Oncology at Monroe Carell Jr. Children's Hospital at Vanderbilt University, a predictive analysis model has been developed that will help providers identify risks of developing blood clots or venous thromboembolisms (VTEs) in pediatric patients. Since hospital-associated VTE cases are ever-increasing, it is becoming more important to identify high risk patients, while the case-controlled studies, many of which remain unvalidated, offer little help. According to Shannon Walker, the principal investigator of the study, “As we noticed that the rate of blood clots was going up and recognized that the adult strategy wasn't going to work for our patients, we wanted to look at each patient's individual risk factors and see how we could focus our attention on targeted blood clot prevention".
The predictive analysis tool draws data from the HER of admitted patients in the hospital while the model itself includes eleven risk factors. The analysis database contained more than 110,000 admissions to Children’s Hospital. The group then validated the tool on more than 44,000 separate admissions. Currently, the efficacy of the tool is being tested in a in a clinical setting, through a trial called Children’s Likelihood of Thrombosis (CLOT). The analysis tool was built with the help of Advanced Vanderbilt Artificial Intelligence Laboratory (AVAIL), which according to Warren Sandberg, MD, PhD, executive sponsor of AVAIL, "served as a catalyst, in this instance by bringing experts in a complex trial development into proximity so that a great synthesis could happen."
Collated by: Debarati Banik, PhD
Genes and Therapy
bluebird bio’s SKYSONA™ gene therapy receives positive opinion from CHMP
On 21st May 2021, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for SKYSONA™ (elivaldogene autotemcel, Lenti-D) for the early treatment of cerebral adrenoleukodystrophy (CALD) in patients under 18 years old with ABCD1 genetic mutations and no matched sibling haematopoietic stem cell (HSC) donor. CALD is a progressive and fatal neurodegenerative disease. The gene therapy introduces a functional ABCD1 gene that can produce the adrenoleukodystrophy protein which breaks down very long-chain fatty acids, toxic accumulations of which cause CALD.
BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics collaborate to discover and develop cell therapies for eye diseases
As part of the alliance, the companies will combine their collective expertise to discover and develop off-the-shelf iPS cell therapies for ocular diseases. Under this agreement, BlueRock Therapeutics will have the option to exclusively license from both parties three (3) retinal cell therapy programs focused on dry age-related macular degeneration (AMD) and inherited retinal diseases (IRDs) containing human retinal pigment epithelial cells and photoreceptor cells, currently in pre-clinical development.
Click here for more on Genes and Therapy
MedNess Business
Onco-News
Agenus and BMS Announce Exclusive Global License for Agenus’ Anti-TIGIT Bispecific Antibody Program
“AGEN1777’s differentiated mechanism of action provides the potential for potent anti-tumor activity; catalyzing our clinical TIGIT strategy aimed at serving more patients with unmet needs in cancer,” said Debbie Law, D.Phil., Senior Vice President, Head of Tumor Microenvironment Thematic Research Center, Bristol Myers Squibb. “We look forward to working with Agenus to develop this important therapy as we continue to combat I-O resistance.”

Calithera Biosciences and Antengene Enter Worldwide License Agreement for Development & Commercialization of CB-708
“This agreement validates the capabilities of our drug discovery engine and represents a significant milestone for our CD73 program,” said Susan Molineaux, PhD, president and chief executive officer of Calithera. “Antengene brings significant enthusiasm and proven global capabilities to the development and future commercialization of CB-708, a potential best-in-class oral small molecule CD73 inhibitor. This licensing agreement enables the continued advancement of this promising program, while allowing Calithera to focus our resources on our more advanced clinical programs evaluating telaglenastat in non-small cell lung cancer and CB-280 in cystic fibrosis.”
BioPharma News
Ginkgo Bioworks and Biogen collaborate over $120 million deal to develop AAV vectors to the cutting edge
Ginkgo Bioworks (Ginkgo; Boston) is a biotechnology company with expertise in genetic and cell engineering to deliver products across industries (from agriculture to pharmaceuticals). Ginkgo recently went public through a merger with a special purpose acquisition company (SPAC) called Soaring Eagle Acquisition Corp., earning a $15 billion evaluation. In the context of the Covid-19 pandemic, Ginkgo offered its robotic cell and genetic programing platform to Moderna to finetune the latter’s mRNA vaccine performance.
On 21st May 2021, Ginkgo and Biogen Inc. (Biogen, Cambridge MA) announced a partnership of $120 million to increase the manufacturability of adeno-associated virus (AAV)-based vectors. For doing this, Biogen will pay $5 million in upfront cash and $115 million as milestone payments to Ginkgo. Ginkgo in turn will use its technological platforms to reprogram the genome of mammalian cell lines and revamp the AAV DNA sequence to increase functional compatibility and drive AAV production to the maximum.
This deal will enable Biogen with its new gene therapy investments in Alzheimer’s and Parkinson’s disease by increasing effective gene delivery to neurons. For Ginkgo, this is an opportunity to prove their capability in gene therapy, an erstwhile unchartered territory, with the potential for massive business opportunities and establishing a strong biotech presence in the future.
Click here for more on mergers, acquisition and business news
Collated by : Richa Tewari, PhD and Divyaanka Iyer
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
Managing Editor
Shalini Roy Choudhury
Genes and Therapy
Managing Editor
Nisha Peter, PhD
Managing Editor
Abhi Dey
Consulting Editor
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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.
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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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