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MedNess: bite-size biopharma and medtech news

14th October, 2020
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Onco I-Analyse
Pfizer’s Ibrance failed to meet primary endpoint in early breast cancer trial
On 9th October, Pfizer announced that Ibrance (Palbociclib) in combination with standard adjuvant endocrine therapy failed to meet the primary endpoint of improvement in invasive disease-free survival (iDFS) in women with HER2neg, HR+ early breast cancer (eBC) with residual invasive disease following neoadjuvant chemotherapy in Phase 3 PENELOPE-B study.
The placebo-controlled trial evaluated 1 year of Palbociclib + 5 years standard adjuvant endocrine therapy in 1,250 pre- and postmenopausal eBC women at high risk (CPS-EG score of ≥3 or 2 with ypN+) of relapse after neoadjuvant chemotherapy. The trial was a collaborative effort between Pfizer, German Breast Group, AGO Study Group, NSABP Foundation, Breast International Group, and many other groups.
The trial did not show any unexpected safety outcomes and detailed results will be presented at an upcoming medical congress.
The investigators will continue to evaluate subgroup data and believe that “key learnings will emerge from the large number of biomarkers being analyzed from collected tumor tissue”. This will strengthen the understanding for future research in early breast cancer with Pfizer’s next-gen CDK2/4/6 inhibitor, PF-06873600.
This is another setback for Pfizer as in May, based on IDMC recommendation, Palbociclib’s Phase 3 PALLAS  trial was stopped after interim analysis for unlikeliness to show significantly improved iDFS as an adjuvant therapy in HER2neg/HR+ eBC. However, Lilly’s Verzenio demonstrated statistically significant results from the Phase 3 MonarchE trial as an adjuvant therapy in 5,637 high-risk women with HER2neg/HR+ eBC and is set for regulatory filings this year.
Currently, Ibrance is the leader in metastatic breast cancer (mBC) with ~79% share of the CDK4/6 class and ~$5B in sales last year, way ahead of Lilly’s Verzenio and Novartis’ Kisqali. However, some analysts speculate that these results might affect Ibrance’s patient-starts in mBC and project Verzenio as a better CDK4/6 inhibitor. Although, many other analysts and Pfizer’s market research with oncologists and KOLs indicated no immediate impact on Ibrance’s strong position in mBC treatment.
Collated by :Shilpa Rawal, PhD
Regulatory News
Update Provided for Lifileucel in Metastatic Melanoma; BLA submission not expected by the end of 2020; anticipated BLA submission in 2021
“TIL is a first-in-class, one-time administration cell therapy targeting solid tumors. As such, definition of the product through a potency assay is an important step toward submission of the BLA,” stated Maria Fardis, Ph.D., MBA, Iovance President and Chief Executive Officer. “We have agreement with the FDA regarding the amount of clinical follow up for the BLA, and we will work closely with the FDA to reach alignment on our assays. Because Iovance recognizes the significant unmet need in the melanoma patient population and believes the compelling clinical data for lifileucel will offer a new therapy for such patients, we are moving ahead with a great sense of urgency. We look forward to further collaboration with the FDA and will provide updates as they become available.”
Nivatrotamab for the Treatment of Patients with Neuroblastoma Granted Orphan Drug Designation and Rare Pediatric Disease Designation by FDA
“We are very pleased with the ODD granted for nivatrotamab, as this potentially would give us seven years of market exclusivity upon market approval. The RPDD makes us eligible for a Priority Review Voucher (“PRV”) upon potential approval of the biologics license application for this rare pediatric cancer. Among our leading compounds, four now have RPDDs, and this designation further increases our chances of ultimately receiving multiple PRVs,” said Thomas Gad, Founder, Chairman and President.
FDA Granted Orphan Drug Designation for CT041 CLDN18.2 CAR-T Cells for the Treatment of Gastric and Gastroesophageal Junction Cancers
“The orphan drug designation of CT041 by the FDA is of great significance to patients with advanced gastric cancer.” said Dr. Zonghai Li, founder, CEO and CSO of CARsgen. “According to the World Health Organization, about 1,030,000 new cases of gastric adenocarcinoma are expected each year [1]. Despite the development of novel therapies, gastric cancer is still a disease with one of the highest unmet medical needs. Our goal is to continue the development of novel, safe and effective immunotherapies. This is our long-standing commitment to cancer patients worldwide.”
Trial Results
Opdivo + Chemo Show Statistically Significant Improvement in pCR as Neoadjuvant Treatment of Resectable NSCLC in Ph 3 CheckMate -816 Trial
“The CheckMate -816 results build on Bristol Myers Squibb’s heritage in the treatment of thoracic cancers, where Opdivo-based regimens have demonstrated superior overall survival in patients with metastatic non-small cell lung cancer and unresectable malignant pleural mesothelioma,” said Abderrahim Oukessou, M.D., vice president, thoracic cancers development lead, Bristol Myers Squibb. “In addition, these data add to our growing scientific understanding of the potential of immunotherapy approaches to transform outcomes in earlier stages of different cancer types, when the immune system may be more responsive. We’re grateful to the patients and investigators who participated in the CheckMate -816 trial.”
Positive Topline Ph 2 Results Announced For Investigational KRAS G12C Inhibitor Sotorasib In Advanced NSCLC
"Targeting KRAS has been a 40-year quest that has left patients with limited options. These topline data underscore our belief in the potential for sotorasib to become the standard of care for non-small cell lung cancer patients with the KRAS G12C mutation who remain in need of new treatment options," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "We now have more than 500 patients who have been enrolled across clinical studies, and we are rapidly moving forward with a broad-based development program, which includes monotherapy studies in non-small cell lung cancer, colorectal cancer and other solid tumors harboring the KRAS G12C mutation, as well as a suite of combination studies.  We look forward to discussing the current results with the U.S. Food and Drug Administration (FDA) and other regulatory agencies, to determine the best path forward for sotorasib as a potential treatment for patients with NSCLC harboring the KRAS G12C mutation."
Click here for more Trial Results
Trial/Program Status
3L cohort added to Ph 2 Pancreatic Cancer Trial; Study opened to Patients Who Have Failed All Approved Standards of Care
“Pancreatic cancer is one of the deadliest forms of cancer, with a five-year survival rate of just five percent, so new and more effective therapies are desperately needed,” said Patrick Soon-Shiong, M.D., Chairman and Chief Executive Officer of NantKwest and ImmunityBio. “By adding the third cohort to this important study, we’re able to enroll patients at all stages of the disease, even those who experience disease progression after the first- or second-line treatment.”
Database for the Ph 3 trial of DCVax®-L for Gliobastoma locked
“We are excited to be so close to the finish line now, after such a long road” commented Linda Powers, the Company’s CEO. “We are hopeful that DCVax®-L can become an important new treatment option for patients who urgently need more and better treatments for Glioblastoma brain cancer.”
Click here for more Trial Status
Collated by : Richa Tewari, PhD
Medness Reviews

Fresenius Announces Plans to Enter Heart, Lung, Organ Failure Markets
Fresenius Medical Care, the dialysis giant has announced a new five-year strategy to expand the reach of its business beyond kidney disease to include treating heart, lung and multi-organ failure.
The company’s renal care is to be bolstered by the development of new therapies using digital technologies, artificial intelligence and big data as well as the use of personalized medicine and home-based care while adding value-based care (transitioning from fee-for-service models to pay-for-performance models), kidney transplantation and new innovations with investments in startups and early-stage companies in the space.
Fresenius will also extend its critical care portfolio to other extracorporeal intensive care therapy areas, including treating heart, lung and multi-organ failure.
A third area in the company’s plan includes expanding its network through acquisitions, partnerships and investments in early-stage startups.
The company said it expects to see annual mid-single-digit percentage increases in revenue, alongside upper-single-digit percentage increases in net income and about a 7% return on invested capital by 2025.

Researchers Aim for a Single Dose Hydrogel Delivery System for The Treatment of Swimmer’s Ear
Researchers from the University of Montana have developed two separate temperature-stable hydrogel delivery systems for the treatment of otitis externa- commonly known as swimmer’s ear.
Although the several treatment options exist on the market for the treatment of ear infections, these drugs are generally ear drops and require frequent doses for the treatment to be effective.
The University of Montana team had now developed a single dose option using two separate delivery systems and have test their efficacy them in a cellular and mouse models. Their study is published in the journal ACS Biomaterials Science & Engineering.
Both systems combine activated tetraethyl orthosilicate with different large molecules to create a hydrogel, which remains in a liquid state inside a syringe, but quickly forms a gel upon entering the ear, allowing for sustained drug release. The hydrogel temperature stable hydrogels are paired with the antibiotic ciprofloxacin
The drug-infused hydrogels managed to wipe out the causal bacteria Pseudomonas aeruginosa and Staphylococcus aureus in cell cultures at doses 100 times lower than those used in most ear drops.
The gels also proved safe when tested on models of human skin and dissipated within 10 days in mouse ears. They had no significant impact on the mice’s hearing compared to standard ear drops.
Currently the only FDA approved single dose treatment for swimmer’s ear is ciprofloxacin otic suspension, which must be refrigerated and prepared before use.
The team hopes to overcome the need for refrigeration due to the temperature stable platform.
Medness Business
Onco-News

Seattle Genetics, Inc. Announces Corporate Name Change to Seagen Inc.
“The change in our corporate name from Seattle Genetics to Seagen reflects who we are today, as we expand our presence beyond the Pacific Northwest and outside of the United States to support the commercialization of TUKYSA globally,” said Clay Siegall , Ph.D., President and Chief Executive Officer of Seagen. “We have three marketed products and a robust development pipeline of novel targeted product candidates. As we increase our global presence by adding new team members and locations outside of the United States as well as through strategic partnerships, we are better positioned to bring important new therapies to cancer patients around the world. Our passion for helping patients is stronger than ever.”
Vincera Pharma, Inc Announces Exclusive License Agreement with Bayer Including a Clinical-stage PTEFb/CDK9 Inhibitor and a Preclinical Bioconjugation Platform
“This license agreement with Bayer creates the foundation of Vincera’s targeted clinical oncology pipeline, with a potentially best-in-class asset, while positioning us for long-term growth across two therapeutic platforms,” said Ahmed Hamdy M.D., Chief Executive Officer of Vincera. “Our lead asset, VIP152, is a small molecule PTEFb/CDK9 inhibitor with very encouraging data from monotherapy Phase 1 studies, including 2 of 7 patients with durable remissions of over 2 years in the very aggressive indication of relapsed/refractory double-hit DLBCL. In addition, preclinical data support our belief that VIP152 is the most selective CDK9 inhibitor in the clinic with on-target depletion of oncogenic MYC and MCL1 mRNA transcripts in patients. These results, combined with the acceptable safety profile seen to date, suggest that VIP152 could be an important new treatment option for patients with MYC- and MCL1-driven malignancies. Importantly, with proof-of-concept clinical data in hand, we are poised to execute on a strategic clinical development plan with the potential for multiple accelerated approvals in the U.S. Expansion of the current Phase 1b study to include these patient populations is expected to begin in 2021.”
BioPharma and MedTech
Bristol Myers Squibb acquires MyoKardia for $13.1 Billion along with the promising Hypertrophic Cardiomyopathy drug Mavacamten
On 5th October 2020, Bristol Myers Squibb acquired MyoKardia, a clinical-stage company for $13.1 billion. This ginormous deal centers around the potential drug mavacamten (MYK-461) designed to treat obstructive hypertrophic cardiomyopathy (HCM). According to Giovanni Caforio, CEO, Bristol-Myers Squibb, Mavacamten will strengthen the cardiovascular portfolio of BMS. Additionally, Mavacamten could be filed with the FDA in the first quarter of next year and eventually launch at the end of next year. The merger deal also ensures BMS’s full right to MyoKardia’s other promising pipeline, including two clinical-stage therapeutics: danicamtiv (formerly MYK-491) and MYK-224.
Takeda and Arrowhead signs $1.04 billion merger deal for Alpha-1 Antitrypsin-Associated Liver Disease treatment 
On 8th October 2020, Takeda announced a collaboration and licensing pact to develop ARO-AAT, an RNAi therapy to treat alpha-1 antitrypsin-associated liver disease (AATLD). The partnership deal between Osaka-based Takeda Pharmaceutical and Pasadena, California-based Arrowhead Pharmaceuticals is worth $1.04B including an upfront payment of $300M and potential development, regulatory and commercial milestones up to $740M. Arrowhead’s first-in-class therapy ARO-AAT will be co-developed and co-commercialized in the United States by both companies and according to the pact they will share a 50/50 profit. Takeda will be entitled to commercialize ARO-AAT outside the U.S according to the exclusive license.
“This agreement also supports our strategy of using partnering selectively to continue to invest in our Targeted RNAi Molecule (TRiMTM) platform and the growing pipeline of RNAi therapeutics targeting diverse tissue types, while focusing our commercial organization on opportunities in two key areas of cardiometabolic and pulmonary.” - Christopher Anzalone, CEO, Arrowhead.
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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