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MedNess: bite-size biopharma and medtech news

30th June, 2020
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HIGHLIGHTS
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FDA rejects Intercept Pharma’s drug for NASH
“At no point during the review did the FDA communicate that OCA was not approvable on an accelerated basis, and we strongly believe that the totality of data submitted to date both meet the requirements of the Agency’s own guidance and clearly support the positive benefit-risk profile of OCA,” said Mark Pruzanski, M.D., President and CEO of Intercept
Collated by :  Ananda Ghosh, PhD and Nisha Peter PhD 
COVID19 Special

WHO, global partners launch $31B COVID-19 pandemic response program
On 26th June 2020, The World Health Organization (WHO) announced an ambitious program along with global partners for providing worldwide pandemic response.Under the $31.3 billion program, WHO and global partners will collaborate under four pillars namely vaccines, therapeutics, diagnostic and the health systems connector.
For the vaccine pillar, WHO along with global groups like Gavi, and the Vaccine Alliance and the Coalition for Epidemic Preparedness Innovations (CEPI), aims to speed the development process for the vaccine in all countries and simultaneously establish manufacturing and distribution infrastructure under an $18 billion program. The group aims to deliver 2 billion COVID-19 vaccine doses by the end of 2021 in countries with the greatest need.
The therapeutics pillar, convened by the Wellcome Trust and UNITAID, aims to develop effective new therapeutics, and ensure the manufacture, procurement, and distribution of 245 million courses of these treatments to low and middle-income countries by mid-2021.
The diagnostics pillar, convened by the Global Fund and the Foundation for Innovative Diagnostics, focuses on developing new simple, affordable, high-quality and rapid diagnostics to detect infection and contain disease spread, with 500 million of these tests reaching people in low and middle-income countries by mid-2021.
The final, Health Systems Connector pillar, led by the World Bank and the Global Fund, is working to ensure that these tools reach the people who need them. Already, the groups have raised $3.4 billion for their products pillar. The commitment leaves a $13.7 billion funding gap of “immediate needs” for the partners to fundraise from donors.
WHO’s campaign comes as leading COVID-19 vaccine candidates race forward, with phase 3 studies for some programs planned this summer. Amid the race, the U.S. and Europe have inked deals to secure supplies should the candidates work, raising concern from some experts that low- and middle-income countries will not be able to secure immediate access.
**Watch this space for more updates**
Drug Approvals
FDA Approves KEYTRUDA® in Recurrent or Metastatic cSCC Patients Not Curable by Surgery or Radiation
“Cutaneous squamous cell carcinoma is the second most common form of skin cancer,” said Dr. Jonathan Cheng, vice president, clinical research, Merck Research Laboratories. “In KEYNOTE-629, treatment with KEYTRUDA resulted in clinically meaningful and durable responses. Today’s approval is great news for patients with cSCC and further demonstrates our commitment to bringing new treatment options to patients with advanced, difficult-to-treat cancers.”
FDA Approves XPOVIO® (selinexor) for the Treatment of R/R DLBCL Patients
“The accelerated approval of oral XPOVIO in patients with relapsed or refractory DLBCL is a significant milestone for the patients and families who currently have limited treatment options available for their disease,” said Sharon Shacham, PhD, MBA, Founder, President and Chief Scientific Officer of Karyopharm. “This approval marks the first for an oral agent for patients with previously treated DLBCL and the first approval of any single drug for this highly aggressive type of lymphoma. Additionally, this is now the second commercial oncology indication for XPOVIO, highlighting its novel mechanism of action, ease of administration and ability to produce rapid and durable responses in patients with heavily pretreated disease. We share this tremendous achievement with the patients, employees, caregivers and physicians who have tirelessly contributed to the advancement of XPOVIO from its original discovery and clinical development to today’s second FDA approval.”
Regulatory News
FDA Advisory Committee meeting to review belantamab mafodotin for the treatment of RRMM patients 
Dr Axel Hoos, Senior Vice President and Head of Oncology R&D, GSK said: “We believe belantamab mafodotin and the results from the DREAMM clinical trial programme have significant potential for patients with relapsed/refractory multiple myeloma who have limited treatment options. We look forward to participating in the upcoming advisory committee meeting and working with the FDA to complete its review of the BLA.”
IMBRUVICA® (ibrutinib) Seeks to Expand U.S. Label with Long-Term Ph 3 iNNOVATE trial Data in Waldenström's Macroglobulinemia (WM)
"Since IMBRUVICA became the first FDA-approved medicine to treat people living with Waldenström's macroglobulinemia more than five years ago, it has significantly changed the treatment landscape for this rare and incurable form of non-Hodgkin's lymphoma," said Danelle James, M.D., M.A.S., IMBRUVICA Global Development Lead, Pharmacyclics LLC, an AbbVie company. "This latest submission reinforces how IMBRUVICA has provided an innovative treatment option for WM patients and our commitment to supporting this patient community."
Trial Results
12-month efficacy and immunological data from Ph 1/2 trial of ONCOS-102 + SOC chemotherapy in malignant pleural mesothelioma (MPM) announced
Dr. Magnus Jäderberg, Chief Medical Officer of Targovax, said: “We are very pleased to see the encouraging first line PFS data holding up in the 12-month analysis, with early signs of positive survival outcomes. We are particularly excited to observe a broad and profound immune activation in the ONCOS-102 treated patients, which confirms the proposed mode of action. ONCOS-102 treatment clearly drives a favorable remodeling of the tumor microenvironment, and this remodeling is linked to better clinical outcomes. This immune activated tumor micro-environment provides the key scientific rationale and an ideal backdrop for combination treatment with a checkpoint inhibitor. These data set us up perfectly to move forward with a trial combining ONCOS-102 and a checkpoint inhibitor, which we believe will release the full potential of immunotherapy in this hard-to-treat patient population”.
Positive Interim Data Presented on VAL-083 Demonstrating Favorable Outcomes in Both 1L and rGBM patients
Dr. John de Groot, Chairman of the Department of Oncology at MD Anderson Cancer Center and planned Principal Investigator for the VAL-083 arm of the GCAR GBM AGILE registration study, noted, "These data continue to demonstrate an improvement over the historical outcomes of standard therapy and validate VAL-083's inclusion in a more robust setting as part of the GBM AGILE study. In MD Anderson's Glioblastoma Moon Shots Program, we are looking to create giant leaps to help patients with GBM where treatment options are limited. It is our hope that VAL-083 may serve as an important new therapy to help physicians and patients dramatically reduce mortality and suffering due to this deadly cancer. We continue to be encouraged by these results and are excited by the opportunity to collaborate with DelMar and GCAR to further explore the potential of VAL-083."
Trial/Program Status
DISCONTINUATION: Vecabrutinib program to be discontinued in R/R CLL and other B-cell malignancies
“Although vecabrutinib continues to exhibit an excellent safety profile, there is insufficient evidence of activity in BTK-inhibitor resistant B-cell malignancies to advance the drug into the planned Phase 2 portion of the trial. One partial remission was observed after 11 treatment cycles in a CLL patient treated in Cohort 5 (300 mg BID) and a number of patients treated across the dose range explored (25 mg to 500 mg BID) saw stable disease; however, no other remissions have been observed,” said Dayton Misfeldt, Interim Chief Executive Officer of Sunesis. “We will complete the Phase 1b and evaluate the best path forward for vecabrutinib. We are grateful for the patients and their families who participated in this trial, as well as the investigators and research staff at our trial sites.”
FDA lifts partial clinical hold placed on Ph 2 TELLOMAK trial of lacutamab (formerly IPH4102) in patients with advanced T-cell lymphomas
“We are pleased that the FDA has approved the new clinical batch for the TELLOMAK trial, and we can resume enrollment of patients with Sézary syndrome and mycosis fungoides given the importance of novel and effective treatment options needed for these patient populations,” commented Pierre Dodion, MD, Executive Vice President and Chief Medical Officer of Innate Pharma. “We’re confident in our ability to supply lacutamab in this important trial moving forward, and look forward to reactivating the trial globally as quickly as possible.”
Click here for more Trial/Program Status
Collated by : Richa Tewari, PhD
MedNess @ HealthIT
Utilization of machine learning in detecting new type of malignant cells in the brain
In a recent study published in eLife, researchers from Vanderbilt university have developed and utilized a novel technology called the Risk Assessment Population IDentification (RAPID) tool to recognize the coordinated patterns of protein expression and modification associated with survival outcomes. RAPID is an open-source machine learning algorithm. The team of researchers used data on cellular proteins that govern the identity and function of neural stem cells and other brain cells through a method called single-mass cytometry, in order to develop the tool. According to Dr. Rebecca Ihrie, associate professor of cell and developmental biology at Vanderbilt, “Without any human oversight, RAPID combed through two million tumor cells – with at least 4,710 glioblastoma cells from each patient – from 28 glioblastomas, flagging the most unusual cells and patterns for us to look into”. The machine learning analysis revealed patterns of protein expression typical of malignant cells, but novel in nature compared to the other known malignant types. The other critical progress was the ability to complete the analysis in an unsupervised manner, relying entirely on the machine learning processes.  Read the full story here. Read the original paper here. 
The route to safer drug-development may be through machine learning
A tool to identify proteins associated with adverse drug side effects has been developed through a collaboration between Harvard Medical School and the Novartis Institutes for BioMedical Research, offering further insight into how the human body responds to drug compounds at the molecular level. This tool is designed to screen drug candidates for potential adverse reactions before they reach the usual track of clinical trials or enter the market as approved medicines. According to the first co-author in the paper, Robert Ietswaart, who belongs to the genetics lab of Stirling Churchman in the Blavatnik Institute at HMS, “Although it cannot predict all possible adverse effects, we hope that our work will help researchers spot potential trouble early on and develop safer drugs in the future.” The team combined an existing database of reported adverse drug reactions and another database of 184 proteins that specific drugs are often known to interact with. They built the algorithm to learn from the existing data and thereby uncovered 221 associations between individual proteins and specific adverse drug reactions, some of which were unknown. The associations were indicative of proteins that may act as drug targets and contribute to specific side effects, as opposed to the other proteins who could be innocent bystanders. With further development in this open source tool, this can be a major breakthrough within the current setting of healthcare saving millions of dollars in clinical trials screening patients for safety and efficacy, and also enable the healthcare system produce a rapid response in times of emergency, such as the current COVID-era. The full study, published in EBioMedicine can be read here.
Collated by :  Debarati Banik
MedNess Reviews
Researchers from UCSD develop a gene therapy approach for the treatment of Parkinson’ disease
An accidental discovery made by a researcher at the University of San Diego, California (USCD) several years back has offers new hopes for the use of gene therapy for treating Parkinson’s disease.
Earlier the researcher had silenced a gene called PTB in mouse fibroblasts. Within weeks, it was observed that most fibroblasts were gone, and the rest had transformed into neurons. Now, researchers in the same lab are applying that discovery to Parkinson’s disease in the hopes of creating a one-time gene therapy to replace the dopamine-producing neurons that are lost to the disease.
The UCSD team has developed a gene therapy technique that silences the PTB gene in mouse models of Parkinson’s. The gene therapy turned astrocytes into dopamine-producing neurons, erasing symptoms of the disease.
Using a viral vector, the team delivered the antisense oligonucleotide for the PTB gene to the midbrain of the mice and compared them with untreated controls. The population of neurons in the brains of mice that received the treatment increase by 30%, and their dopamine levels returned to normal. The control mice saw no improvements.The team reported that within three months of the gene therapy, the treated mice were able to move their limbs normally, and they remained free of Parkinson’s symptoms for the rest of their lives.
Gene base therapies for neurodegenerative disorders are being developed by several companies. Biogen’s Spinraza to treat spinal muscular atrophy is an antisense oligonucleotide drug, as are many experimental medicines. Earlier this month, Eli Lilly formed a $20 million deal with Evox Therapeutics, which is using the approach to develop treatments for Duchenne muscular dystrophy and several neurological disorders.Startup Prevail Therapeutics is also developing a gene therapy to treat Parkinson’s that’s caused by mutations in the GBA1 gene.
Medness Business
Onco-News

Gilead Sciences Secures Exclusive Option to Acquire Pionyr Immunotherapeutics
“Pionyr is pursuing promising, novel biology in the field of immuno-oncology,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. “The agreement represents important progress as we continue to build out Gilead's presence in immuno-oncology with innovative and complementary approaches. We look forward to seeing the programs advance with the goal of developing new therapies that will improve the treatment of cancer.”
Collated by : Richa Tewari, PhD
Editors' Desk
Richa Tewari, PhD
Oncology News
Esha Sehanobish, PhD
MedNess Plus
Arundithi Ananthanarayanan
MedNess Reviews
Debarati Banik
HealthIT
Divyaanka Iyer
MedNess Reviews
Nisha Peter, PhD
Managing Editor
Mayur Vadhvani, PhD
Consulting Editor
Abhi Dey
Consulting Editor
Ananda Ghosh, PhD
Founder
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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 , Mayur Vadhvani and Abhinav Dey 
Concept and Design: Ananda Ghosh and Nisha Peter
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