View this email in your browser

MedNess: bite-size biopharma and medtech news

9th June, 2020
Subscribe here
MedNess This Week
HIGHLIGHTS
Our Sponsors
COVID Special

Gilead announces topline results from the Phase 3 trial of Remdesivir in hospitalized patients with moderate Covid-19 pneumonia
Gilead Sciences recently announced results from their Phase 3 SIMPLE trial of Remdesivir in hospitalized Covid-19 patients with pneumonia. Remdesivir is an investigational drug that has not been approved by FDA yet for any use. Its safety and efficacy are not yet known.
“We now have three randomized, controlled clinical trials demonstrating that remdesivir improved clinical outcomes by several different measures. Today’s results showed that when treating moderate disease, a 5-day course of remdesivir led to greater clinical improvement than standard of care, adding further evidence of remdesivir’s benefit to previously released study results. The National Institute of Allergy and Infectious Diseases’ placebo-controlled study showed that remdesivir enabled more rapid recovery and that earlier treatment improved clinical outcomes. Our SIMPLE-Severe study showed that when treating patients with severe disease, 5 days of remdesivir led to similar clinical improvements as a 10-day course,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. “The additional data we have in hand today will further guide our research efforts, including evaluating treatment earlier in the course of disease, combination studies with other therapies for the most critically ill patients, pediatric studies and the development of alternate formulations.”
AstraZeneca makes progress towards equitable global access to the University of Oxford’s Covid-19 vaccine
AstraZeneca is moving ahead by broadening the global access to the University of Oxford’s Covid-19 vaccine. It has entered into important landmark deals and agreements with the Coalition for Epidemic Preparedness (CEPI), Gavi the Vaccine alliance and the Serum Institute of India (SII).
Pascal Soriot, Chief Executive Officer, AstraZeneca, said: “We are working tirelessly to honour our commitment to ensure broad and equitable access to Oxford’s vaccine across the globe and at no profit. Today marks an important step in helping us supply hundreds of millions of people around the world, including to those in countries with the lowest means. I am deeply grateful for everyone’s commitment to this cause and for their work in bringing this together in such a short time.”
Click Here for more details and updates on COVID19
Collated by : Esha Sehanobish, PhD
Drug Approvals
BRUKINSA™ (Zanubrutinib) Approved in China for Patients with R/R CLL or SLL and R/R MCL
“The concurrent approvals of BRUKINSA in R/R CLL/SLL and R/R MCL are a tribute to the collective expertise and hard work of the BeiGene team. With two product approvals covering four indications in China and one in the United States in merely seven months, we continue to focus on execution in advancing our broad portfolio,” commented John V. Oyler, Co-Founder, Chief Executive Officer, and Chairman of BeiGene.
European Commission approves Sarclisa® (isatuximab) for RRMM Patients
“The EC approval of Sarclisa represents an important additional therapeutic option and may set a new standard of care for myeloma patients in Europe who are in need of new effective treatments because their disease has returned or they have become refractory to their previous treatment,” said John Reed, M.D., Ph.D., Global Head of Research and Development at Sanofi. “Sarclisa in combination with pom-dex demonstrated median progression-free survival of nearly one year, a five-month improvement over pom-dex alone, in patients who had already failed at least two prior therapies.”
Regulatory News
Margetuximab Granted Orphan Drug Designation in the U.S. for Gastric Cancer
“We are pleased that the FDA has granted orphan status to margetuximab,” said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. “We believe that our immune-enhancing antibody targeting HER2 has the potential to improve upon the clinical activity of existing standard of care for patients with gastric or gastroesophageal cancer.”
FDA Accepts BLA for Danyelza™ (naxitamab) for the Treatment of Neuroblastoma for Priority Review; PDUFA: 30Nov2020
“We believe that the FDA’s acceptance of our BLA for priority review of our first leading antibody compound, Danyelza (naxitamab), is a significant achievement for Y-mAbs and a crucial step forward as we anticipate that Danyelza, if approved, can address a significant unmet medical need for children with relapsed/refractory high-risk neuroblastoma,” stated Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy. 
FDA Accepts Filing of an NDA for Tivozanib in R/R RCC patients; PDUFA: 31Mar2021
“The acceptance of our NDA filing marks yet another important milestone for AVEO, as we pursue our goal of providing RCC patients whose disease has relapsed or become refractory to multiple lines of therapy with a meaningful new treatment option,” said Michael Bailey, president and chief executive officer. “We look forward to working closely with the FDA over the coming months during their review of our application. In parallel, we continue to focus on commercial-readiness to ensure we are well positioned to support the potential launch of tivozanib, subject to approval.”
Lynparza (olaparib) recommended for approval in EU by CHMP for the 1L maintenance treatment of gBRCAm metastatic pancreatic cancer patients
José Baselga, Executive Vice President, Oncology R&D, said: “Patients with advanced pancreatic cancer have seen limited treatment advances over the last few decades. We are now one step closer to bringing the first targeted medicine to certain biomarker-selected patients with advanced pancreatic cancer in the EU.”
Trial Results
New interim data announced from Ph 2 part of Ph 1/2 trial of RP1 + Opdivo® in CSCC and anti-PD-1 refractory melanoma
“CSCC is a significant commercial opportunity that we believe has the potential to drive substantial value for the company. The number of complete responses (CRs) observed is highly suggestive that our combination approach with RP1 can provide better patient outcomes compared to anti-PD-1 therapy alone, where CRs are infrequent,” said Philip Astley-Sparke, CEO of Replimune.  “In anti-PD-1 refractory melanoma, we believe we also have a strong efficacy signal and are optimistic that our currently-enrolling 125 patient cohort could generate data to support regulatory approval, pending feedback from the U.S. Food and Drug Administration (FDA) and other regulatory agencies. We are also excited to be moving to evaluate RP1 in anti-PD-1 refractory non-small cell lung cancer (NSCLC), given the large unmet need in this tumor type. We believe we have established clinical proof of principle with RP1 in immune-responsive tumor types and in anti-PD-1 refractory cancers, and now have a solid foundation upon which to establish our product candidates more broadly as the second cornerstone of immuno-oncology.”
Sarclisa® (isatuximab) combination therapy demonstrated superior PFS and clinically meaningful depth of response in RRMM patients
“In the Phase 3 IKEMA trial, the addition of Sarclisa to carfilzomib and dexamethasone reduced the risk of disease progression or death by 47 percent compared to treatment with carfilzomib and dexamethasone alone,” said Philippe Moreau, M.D., Department of Hematology, University Hospital of Nantes, France. “These results suggest the potential of Sarclisa to become a new standard of care in the relapsed multiple myeloma setting.”
Preliminary data announced from first 10 patients in safety cohort of Ph 2 ILLUMINATE-206 trial of tilsotolimod + Opdivo® (nivolumab) and Yervoy®* (ipilimumab) in IO-naive MSS-CRC patients
“We are encouraged by the initial safety profile of this first-time triplet combination,” stated Elizabeth A. Tarka, M.D., Idera’s Chief Medical Officer. “We look forward to continuing to explore the potential clinical benefit of tilsotolimod in combination with ipilimumab and nivolumab in MSS-CRC, possibly yielding a treatment alternative for these patients with few current options.”
Preliminary Results of IBI310 in Ph 1 Trial Announced
Dr. Hui Zhou, Vice President of Medical Science and Strategic Oncology of Innovent, said: "CTLA-4 is an important immunosuppressive receptor, and there are a number of CTLA-4 related clinical studies on-going both in domestic and abroad, while currently only one has been approved. IBI310 is the CTLA-4 monoclonal antibody of fastest progress in China. The preliminary clinical results of IBI310 in combination with sintilimab show acceptable safety and anti-tumor activity, suggesting a synergistic enhancement effect. Currently, Phase 2/3 clinical studies of IBI310 in combination with sintilimab are on-going in multi tumors. We hope to evaluate the clinical results of IBI310 in combination with sintilimab and bring this therapy to more patients in need as soon as possible. "
Trial/Program Status
Ph 2 trial announced to evaluate PDS0101 + SoC CRT for treatment of locally advanced cervical cancer
“We are pleased to announce this Phase 2 clinical trial being performed to further validate our novel Versamune®-based immunotherapy platform and lead asset PDS0101,” said Dr. Frank Bedu-Addo, CEO of PDS Biotech. “We look forward to investigating PDS0101 as a potentially safe and effective immunotherapeutic combination with standard of care chemoradiotherapy to improve the treatment options for women with locally advanced cervical cancer.”
DSMB Recommendation for Plinabulin is to Continue NSCLC Ph 3 Dublin-3 Study Without Modification
“The Dublin-3 study was designed to quantify the immune-enhancing potential of Plinabulin,” said Dr. Ramon Mohanlal, BeyondSpring’s Chief Medical Officer and Executive Vice President, Research and Development. “We selected tumors with a measurable lung lesion (per RECIST 1.1), which has more TMB (tumor mutation burden) to prospectively test this population (which had encouraging results in our Phase 2 analysis). This population – over 70 percent of EGFR wild-type NSCLC patients – represents new subclones with a higher probability of harboring antigens capable of stimulating the immune system (Mohanlal, ESMO 2019; Mohanlal, IASLC / World Conference on Lung Cancer, 2019). The DSMB’s green light is an encouraging step as we continue this trial without modifications following our second interim dataset review.”
Full Enrollment of Ph 3 Trial of eprenetapopt Completed in TP53 Mutant MDS patients
“Completion of enrollment in this Phase 3 trial is an important milestone for Aprea and our first-in-class p53 reactivator, eprenetapopt,” said Christian S. Schade, President and Chief Executive Officer of Aprea. “We continue to advance the development of eprenetapopt with the goal of providing an urgently needed therapeutic option to patients with p53 mutated MDS.”
MedNess Reviews
FDA Approves Antibiotic to Treat Hospital-Acquired Bacterial Pneumonia and Ventilator-Associated Bacterial Pneumonia
RECARBRIO (a combination of imipenem-cilastatin, and relebactam) developed by Merck has recently been green-lighted by the FDA for the treatment of patients over 18 years of age, contracted with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP). HABP and VABP are often a result of infection with Gram-negative microorganisms: Acinetobacter calcoaceticus-baumannii complex, Enterobacter cloacae, Escherichia coli, Haemophilus influenzae, Klebsiella aerogenes, Klebsiella oxytoca, Klebsiella pneumoniae, Pseudomonas aeruginosa and Serratia marcescens. The infections cause symptoms ranging from fever, chills, cough, chest pain, and increased oxygen requirements.
 In a Phase III RESTORE-IMI2 trial of 535 hospitalized adults with HABP/VABP, when compared with piperacillin/tazobactam, RECARBRIO met the primary and critical secondary endpoints, demonstrating non-inferiority to PIP/TAZ in terms of preventing mortality through Day 28 of the study. The most common side effects of RECARBRIO included anemia, diarrhea, hypokalemia, hyponatremia and increased aspartate/alanine aminotransferase levels. Patients receiving treatment with RECARBRIO should also be pre-screened for hypersensitivity to carbapenems, penicillins, cephalosporins, other beta-lactams, and other allergens, as well as for other central nervous system disorders.
 The application was granted a Qualified Infectious Disease Program (QIDP) designation, intended for products that treat life-threatening infections under the Generating Antibiotic Incentives Now (GAIN) title of the FDA Safety and Innovation Act, with a Fast Track and Priority Review designation.
Collated by : Tanmoy Samaddar
Medness Plus
FDA approves artesunate for injection as the only drug in the US to treat severe malaria
FDA recently approved artesunate for injection to treat patients with severe malaria as the only drug in the US to serve this purpose. The administration will be intravenous with a complete treatment course of a required oral antimalarial regimen. The application for artesunate was granted a Priority Review designation and an Orphan drug designation to Amivas.
“This approval will now give patients more access to a lifesaving drug,” said John Farley, M.D., acting director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research. “Furthermore, the risk of developing severe malaria emphasizes the importance of taking medications to prevent malaria and using mosquito avoidance measures when traveling to malaria-endemic areas.”

FDA approves VESIcare LS, the first treatment for a form of bladder dysfunction in children ages 2 years and older
FDA recently approved the first treatment for neurogenic detrusor overactivity (NDO), a form of bladder dysfunction associated with neurological impairment in pediatric patients 2 years and older. The approved treatment is VESIcare LS (solifenacin succinate) oral suspension which will be taken by mouth. VESIcare was initially approved for the treatment of overactive bladder in adults 18 years and older, back in 2004. The approval was granted to Astellas Pharma US, Inc.
“This is the first FDA-approved treatment for NDO patients as young as two years of age,” said Christine P. Nguyen, M.D., acting director, FDA’s Division of Urology, Obstetrics and Gynecology, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, Center for Drug Evaluation and Research. “In addition, prior to today’s approval, the current standard of care for many of these patients required up to three times a day dosing, and this treatment requires only once a day dosing.”
Click Here for more on MedNess Plus
Collated by : Esha Sehanobish, PhD
Medness Business
Onco-News

AstraZeneca to discover and develop novel therapies targeting RNA-modifying proteins in oncology collaboration with Accent Therapeutics
José Baselga, Executive Vice President, Oncology R&D, AstraZeneca said: “The promise of RMP inhibition is a compelling area of exploration for AstraZeneca. With this collaboration, we will seek to identify novel targets and unlock the full potential of our medicines. We believe that the Accent team’s expertise in RNA-modifying protein biology and drug discovery complements AstraZeneca’s extensive research and development portfolio.”
Jounce Therapeutics Regains Worldwide Rights to JTX-8064 from Bristol Myers Squibb
“We are thrilled to regain the rights to JTX-8064 and we view this as a significant opportunity for Jounce. Though we highly valued our longstanding partnership with Celgene, now a Bristol Myers Squibb company, having an additional wholly-owned program enables us to further our mission to discover new immunotherapies from a variety of important immune cell types, and develop them for patients who are not well served by today’s therapies,” said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. “The discovery and development of JTX-8064 showcases the strength of our Translational Science Platform in target identification, and our ability to move programs towards the clinic in a rapid manner. In particular, we believe that LILRB2 may function as an immune checkpoint for macrophages and based on our body of existing preclinical data, JTX-8064 has the potential to re-program tumor-associated macrophages within the tumor microenvironment and enhance anti-tumor immunity. We are eager to advance this program into the clinic and will make every effort to do this expeditiously."  
Collated by : Richa Tewari, PhD
Bio-Pharma and MedTech
Why AstraZeneca’s merger talks with Gilead may not take off
On 8th June 2020, Bloomberg carried a report of a potential merger proposal extended to Gilead Sciences by AstraZeneca. The details of the proposal are unavailable. There is, however, skepticism over the merger taking place as both parties are vested in their own drug pipelines with different strategies.
“We do not believe a merger with AZ is inline w/GILD’s current approach to BD, preferring smaller deals. There could be potential onc. & inflammation synergies, but otherwise is not a rational combo.”- Evan Seigerman (Credit Suisse)
“From a biopharma industry point of view, it may suggest that AZN is looking to leverage its valuation and current strategic position by making a large acquisition to diversify away from their dependence on its blockbuster oncology franchise, and add another strong franchise or two that offsets any risk that may emerge now or in the future in the oncology business.”- Geoffrey Porges (SVB Leerink)
“While interesting and good for GILD’s stock price, we do not view this deal as likely. As we have noted before, we think GILD believes its HIV biz is very underappreciated by the Street and would prefer to build value over time and do its own tuck-in deals”- Michael Yee (Jefferies).
Legend Biotech makes the largest IPO offering of the year at $424 million basis its multiple myeloma CAR-T cell therapy
Legend Biotech Corporation (Legend; Somerset, New Jersey), formed as a china-based subsidiary of GenScript (Piscataway, New Jersey) in 2014, has developed a first in class B cell maturation antigen (BCMA)- targeted CAR-T cell therapy for multiple myeloma. Called JNJ-4528, the efficacy of the treatment was proven in Phase I clinical trials where 25 out of 29 subjects showed complete response to the drug. The trials were conducted in collaboration with Jannsen Biotech (HQ: Horsham, Pennsylvania). JNJ-4528 is now poised for Phase II testing.
On 5th June 2020, Legend announced $23/share in its initial public offering of 18 million shares, giving the IPO a size of $424 million. The IPO was supplemented by the purchase of 1 million shares by GenScript which continues to be the majority shareholder, adding $24 million to Legend’s accounts.
Legend plans to apply for regulatory approval in the US and EU by the third quarter of the year and is also looking at approval for its therapy in Japan. The company plans to utilize $160-180 million in furthering development of JNJ-4528, $60-75 million towards developing manufacturing facilities and $15-20 million for commercialization of the drug, if approved.
Click Here for more news and details on mergers, acquisitions and business updates
Collated by : Divyaanka Iyer
Editors' Desk
Richa Tewari, PhD
Oncology News
Esha Sehanobish, PhD
MedNess Plus
Arundithi Ananthanarayanan
MedNess Reviews
Tanmoy Samaddar
MedNess Reviews
Divyaanka Iyer
BioPharma News
Debarati Banik
HealthIT
Mayur Vadhvani, PhD
Consulting Editor
Abhi Dey
Consulting Editor
Nisha Peter, PhD
Managing Editor
Ananda Ghosh, PhD
Founder
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 : Pixabay
Images : Twitter 
Content Editors: Richa Tewari , Esha Sehanobish , Divyaanka Iyer, Arundithi Ananthanarayanan, Debarati Banik, Tanmoy Samaddar, Mayur Vadhvani and Abhinav Dey 
Concept and Design: Ananda Ghosh and Nisha Peter
Our mailing address is:
MedNess
2160 Matthews Avenue 
Bronx 10462, NY 
NY

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