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MedNess: bite-size biopharma and medtech news

10th November, 2020
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MedNess This Week
HIGHLIGHTS
Our Sponsors
Onco I-Analyse
US FDA lifted the clinical hold on Ph 1 study of P-PSMA-101 in mCRPC
On 2nd November, Poseida Therapeutics announced that the US FDA has lifted clinical hold from the Phase 1 study evaluating P-PSMA-101 CAR-T cells in patients with metastatic Castration-Resistant Prostate Cancer (mCRPC).
Following the announcement, Poseida will immediately resume the trial with protocol modifications relating to increased patient compliance and safety. Some of these amendments will include revised inclusion and exclusion criteria, frequent monitoring and laboratory testing for enrolled patients.
Patient dosing in the trial was initiated in May 2020. However, in August, the FDA had placed clinical hold on the trial following the death of a patient. The patient had progressed on several anti-cancer treatments before receiving P-PSMA-101 and presented no AEs following 7 days of treatment. However, the patient missed the Day 10 and 14 follow-up visits, got hospitalized during that period and died on Day 19 due to hepatic failure.
Although the direct cause of hepatic failure was not determined, the patient had symptoms consistent with macrophage activation syndrome, a serious CAR-T linked overactivation of the immune system. The patient also developed other complications, requiring further investigation of the death.
While CAR-T therapies have CAR T-cells have revolutionized the treatment of relapsed and refractory acute lymphoblastic leukemia and В-cell NHL, demonstration of clinical benefit in solid tumors remained a challenge.
P-PSMA-101, company’s first CAR-T for solid tumors, is an autologous CAR-T therapeutic candidate targeting prostate-specific membrane antigen (PSMA) expressed on mCRPC cells. It was developed using the company’s proprietary piggyBac DNA Modification System, which manufactures cell-based therapeutics with high percentage of stem cell memory T (TSCM) cells. The high percentage of TSCM cells is anticipated to address challenges linked with earlier-generation CAR-Ts.
Collated by :Shilpa Rawal, PhD
Drug Approval
Lynparza approved in the EU as 1L maintenance treatment with bevacizumab for HRD-positive advanced ovarian cancer
Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: “Half of all newly diagnosed patients with advanced ovarian cancer have HRD-positive tumours. Women treated with Lynparza in combination with bevacizumab in the PAOLA-1 Phase III trial lived progression free for a median of more than three years, showing that HRD testing should be an essential component of clinical diagnosis. HRD status can help physicians select a personalised 1st-line treatment regimen for patients to substantially delay relapse in this devastating disease.”
EU Approves Opdivo (nivolumab) + Yervoy (ipilimumab) with Two Cycles of Chemotherapy for 1L EGFR/ALK WT NSCLC
“With a complex disease like metastatic non-small cell lung cancer, the availability of different treatment options is critical for patients, who have diverse needs and challenges,” said Martin Reck, M.D., Ph.D., CheckMate -9LA study investigator, Lung Clinic Grosshansdorf, German Center of Lung Research. “In the CheckMate -9LA trial, combining nivolumab and ipilimumab with two cycles of chemotherapy resulted in clinically meaningful overall survival benefits, which were consistent across patients with non-small cell lung cancer, regardless of PD-L1 expression levels or tumor histologies. Following today’s approval, clinicians in the EU will be able to offer patients a new option that may help achieve early disease control and improve survival.”
Lynparza approved in the EU for mCRPC patients with BRCA1/2 mutations
Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: “The PROfound Phase III trial showed Lynparza provided a clinical benefit for men living with BRCA1/2-mutated metastatic castration-resistant prostate cancer, offering an important option to improve overall survival for these patients in the EU. MSD, along with our collaborator AstraZeneca, looks forward to making this targeted treatment available for patients across the EU as quickly as possible.”
Regulatory News
FDA Clears IND Application for EG-70 for the treatment of NMIBC patients
"The FDA's clearance of our IND for EG-70 is a significant moment in the field of gene therapy, and importantly, a major milestone for patients, as we are now one step closer to a treatment that is so critically needed," said Jason Hanson, President and Chief Executive Officer of enGene. "This milestone is a remarkably exciting one for us as we embark on our next chapter as a clinical stage company, harnessing the power of our non-viral gene therapy platform to impact the lives of patients around the globe."
Trial Results
Ph 3 SEAL Study Meets Primary Endpoint of Significant PFS Increase in Patients with Unresectable Dedifferentiated Liposarcoma
"We are delighted to share these significant top-line results from the Phase 3 portion of the SEAL study, the first, late-stage clinical data for XPOVIO in a solid tumor indication," said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. "The top-line results from the SEAL study are particularly encouraging as advanced dedifferentiated liposarcoma represents a very difficult to treat cancer with no established standard of care and limited treatment options available to patients. XPOVIO may be particularly promising as it represents the first oral therapy to show activity in patients with previously treated liposarcoma. We look forward to presenting the detailed results at the upcoming CTOS Annual Meeting and plan to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the first quarter of 2021 requesting the approval of XPOVIO to treat the patient population studied in SEAL. If approved, XPOVIO would represent the first oral, non-chemotherapy agent available for patients with dedifferentiated liposarcoma. The encouraging data from the SEAL study also provide additional rationale for advancing the clinical development of XPOVIO in other solid tumor indications, including in endometrial, glioblastoma, lung and other cancers where Karyopharm is currently conducting clinical studies."
Interim Results from Ph 2 SUMMIT Trial of Neratinib for EGFR Exon 18 Mutated mNSCLC Announced
Alan H. Auerbach, CEO and President of Puma Biotechnology, added, “We are very pleased with the preliminary activity seen with neratinib in this cohort of patients with EGFR exon 18 mutated NSCLC who have previously been treated with EGFR targeted tyrosine kinase inhibitors. We believe that there is a need for new treatments for these patients and we look forward to the further development of neratinib in this patient population.”
First Complete Remission Announced in Ongoing APVO436 Ph 1 Clinical Trial
"We are greatly encouraged by the complete remission in the patient in cohort 6, which is a wonderful outcome for them," said Marvin White, President and CEO of Aptevo Therapeutics. "We are now in a critical phase of the study, as pharmacokinetic modelling suggests that dosing in cohorts 5 through 8 is in a therapeutic range, which could result in potential clinical activity of the drug. We look forward to continuing the dose escalation and monitoring potential clinical responses as we advance through the upcoming dose cohorts," concluded Mr. White.
Trial/Program Status
Ph 3 trial of Epcoritamab in R/R DLBCL Patients to be initiated
“In collaboration with AbbVie, we have planned a broad, expansive, accelerated epcoritamab clinical development plan to maximize the potential of this promising bispecific antibody, with the ultimate goal of bringing new differentiated treatment options as soon as possible to patients. We look forward to the data from this first Phase 3 trial, especially for relapsed or refractory DLBCL patients as it remains an area of high unmet medical need,” said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.
DESTINY-Breast05 Head-to-Head Ph 3 Trial of ENHERTU® vs T-DM1 Initiated in Patients with HER2+ve Early Breast Cancer at High Risk After Neo-adjuvant Therapy
“Despite recent improvements and approvals of new medicines, there remain significant clinical needs for patients with HER2 positive early breast cancer with residual invasive disease after completing neo-adjuvant treatment. We recognize the important opportunity that exists post-surgery to slow disease progression with further adjuvant treatment,” said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. “This research builds on the data from DESTINY-Breast01 which showed durability of response in previously treated HER2 positive metastatic breast cancer. DESTINY-Breast05 will evaluate ENHERTU in patients with early HER2 positive breast cancer, versus T-DM1, the current standard of care, which marks the first time we will evaluate the clinical benefit of ENHERTU in early breast cancer, reflecting our commitment to transforming treatment for even more patients with HER2 targetable disease.”  
Click here for more Trial Statuses
Conference Coverage - ASH 2020
  1. AbbVie to present blood cancer data from nearly 40 abstracts, including 10 oral presentations, across 11 cancer types 
  2. Acceleron Announces REBLOZYL® (luspatercept-aamt) data analyses from Ph 3 MEDALIST and BELIEVE trials
  3. ADC Therapeutics to present data on pyrrolobenzodiazepine (PBD)-based ADCs – loncastuximab tesirine (Lonca), camidanlumab tesirine (Cami) and ADCT-602
  4. Affimed N.V. announced two abstracts highlighting data related to AFM13
  5. Agios to Present Broad Set of Clinical and Translational Data for Oncology and Rare Genetic Disease Programs 
  6. Allogene Therapeutics Announces Oral Presentation of Initial Results from its Ph 1 Dose Escalation Study of ALLO-715 in RRMM patients
  7. ALX Oncology to present both ALX148 Ph1b ASPEN-01 clinical and preclinical results
  8. Aptose to Present CG-806 and APTO-253 Data
  9. Arcellx announced clinical data from ongoing Ph 1 Study of CART-ddBCMA in RRMM patients
  10. BeiGene to present clinical data on BTK inhibitor BRUKINSA® (zanubrutinib) and non-clinical data on anti-PD-1 antibody tislelizumab in an oral presentation and four posters 
  11. Biosight to Present Updated Clinical Data from Ongoing Ph 2b Study of Aspacytarabine (BST-236)
  12. Cellectis announced one oral presentation of initial data for BALLI-01 clinical trial and one Trials in Progress poster presentation of AMELI-01 clinical trial
  13. Celyad Oncology SA announced presentations on anti-BCMA allogeneic CAR T candidate, CYAD-211, and autologous NKG2D receptor-based CAR T candidates CYAD-01 and CYAD-02
  14. Chi-Med to present initial analysis of the first in human HMPL-689 Ph 1 dose escalation study
  15. Fate Therapeutics announced four oral and eight poster presentations for the induced pluripotent stem cell (iPSC) product platform
  16. Genmab announced more than 40 abstracts including data from Ph I/II trial of epcoritamab in B-cell NHLS, updates on multiple daratumumab clinical trials, teclistamab and talquetamab
  17. Gilead Sciences and Kite announced 16 abstracts, including three oral presentations from the companies’ combined immuno-oncology research and development programs
  18. Incyte to present numerous abstracts, including 7 oral presentations, highlighting data for ruxolitinib, parsaclisib, tafasitamab and ponatinib 
  19. Janssen Pharmaceutical Companies of Johnson & Johnson announced today more than 35 company-sponsored studies, including 10 oral presentations
  20. Jazz Pharmaceuticals announced 11 company-sponsored abstracts, 2 abstracts from collaboration trials with MD Anderson, 1 from a cooperative group trial and 4 from investigator-sponsored trials
  21. Legend Biotech Announces Data Presentations for Ciltacabtagene Autoleucel (cilta-cel), an Investigational BCMA CAR-T Cell Therapy in Development for RRMM Patients
  22. Loxo Oncology at Lilly to present data from the LOXO-305 development program
  23. MacroGenics announced six clinical and preclinical abstracts related to AML and flotetuzumab and one abstract related to tebotelimab
  24. Mustang Bio to present interim Ph 1/2 data on MB-106 in R/R NHL patients
  25. NexImmune to Highlight Preliminary Results from Ph 1/2 Trial of NEXI-001 via Oral Presentation
  26. Roche to present new data across 16 blood disorders 
  27. Sanofi to present new clinical and health-related quality of life data in multiple myeloma and rare blood disorders
  28. Seagen to present multiple ADCETRIS® (brentuximab vedotin) data presentations including 5-year updates from the Ph 3 ECHELON-1 and ECHELON-2 trials in 1L HL or PTCL
  29. Sierra Oncology to Report Momelotinib Long-term OS Data
  30. Syros Pharmaceuticals to present new clinical data from ongoing Ph 2 trial of SY-1425
  31. Takeda to present 22 company-sponsored identifying unique approaches in advancing the treatment of hematologic cancers
  32. TG Therapeutics to present updates from the UNITY-NHL and UNITY-CLL trials
  33. Xencor to present data from the ongoing Ph 1 dose-escalation study of vibecotamab (XmAb®14045) in R/R AML patients
Collated by : Richa Tewari, PhD
Medness @ HealthIT
Robotics coming to aid COVID-associated food insecurity
Food insecurity among students who rely on their schools’ free breakfast and lunch programs has posed a tremendous challenge after the school closures during COVID-era. The robotics institute in the Carnegie Mellon University has set an example of using ML (machine learning) tools to identify routes for food delivery to the families that need it the most, via a program called Carnegie Mellon’s Metro21: Smart City Institute. The program developed cost-effective bus routes for nonprofit organizations, such as, Allies for Children, United Way of Southwestern Pennsylvania, and the Greater Pittsburgh Community Food Bank, to deliver meals to senior citizens, as well as K-12 students and families. The program has been able to deliver 6000+ prepared meals every month since July, by avoiding the traditional routes that required the bus to stop every couple of blocks. For example, in the Penn Hill township of Pennsylvania, almost 4,000 students who rely on free breakfast and lunch at school were identified through a data sharing partnership between the Penn Hills School District and the Allegheny County Department of Human Services. CMU researchers then gathered anonymized address information and fed it to the ML program to locate routes to reach out.
Antibiotic resistance now targeted via ML
Exhibiting a growing threat to world health, antibiotic resistance also adds up to unnecessary costs to the healthcare systems. Antibiotic resistance towards UTI with women persists as one such example. Most physicians tend to prescribe fluoroquinolones due to its lower cost and general effectiveness. However, a significant amount of life-threatening risk remains in the form of secondary infections (e.g., C. difficile and staph) and higher incidents of tendon injuries and aortic tears. Medical associations therefore have set guidelines to use this antibiotic as a second line of treatment instead of primary, which is, unfortunately, often overridden by the overworked clinicians. Recently, Brigham and Women’s Hospital and Massachusetts General Hospital have utilized the EHR from 10,000 women to generate a predictive model which can make a recommendation on whether the UTI condition in the said patient can be treated with first- or second-line antibiotics, and is predisposed to first-line agent as frequently as possible. The researchers from MIT have been instrumental in developing the ML tool capable of making the clinical recommendations, even when the primary causative bacteria are not identified in the UTI patient. Statistically, the ML algorithm was able to reduce the use of secondary line of treatment in 67% of cases, reaching a treatment efficacy of almost 90%, as well as suggested the correct first line antibiotics more accurately than clinicians.
Medness Business
Onco-News

Apollomics, Inc. Closes $124 Million Series C Financing supporting the development of APL-101 and APL-106 programs
“Apollomics is focused on a precision medicine approach that targets specific mutations, amplifications and resistance mechanisms to bring transformative therapies to cancer patients, said Guo-Liang Yu, PhD, Co-Founder, Chairman and Chief Executive Officer. “We appreciate the profound level of support and interest we received during this financing and welcome several new investors to our shareholder base. With this infusion of capital, we will continue our ambitious plans to progress our current pipeline and expand our programs globally.”
Merck to Acquire VelosBio
“Merck is a recognized leader in oncology, and this acquisition reflects the hard work and commitment of all the employees at VelosBio in advancing the science of ROR1,” said Dave Johnson, founder and chief executive officer at VelosBio. “We are very pleased that Merck has recognized the value of our first-in-class ROR1-directed investigational therapeutics. As part of Merck’s oncology pipeline, our lead product candidate, VLS-101, is now well positioned to achieve its maximum potential to benefit appropriate cancer patients in need.”
BioPharma and MedTech
Decibel Therapeutics secured $82 M Series D Funding to strengthen hearing loss gene therapy research
On November 9, 2020 Decibel Therapeutics, Boston based biotech company raised $82 Million series D funding. The financing was led by OrbiMed, with new investments from BlackRock Health Sciences, Casdin Capital, Janus Henderson, Samsara BioCapital, and Surveyor Capital and Existing investors including Foresite Capital, GV, S-Cubed Capital, Sobrato Capital, SR One, Third Rock Ventures, and other institutional investors. Decibel Therapeutics, currently have multiple pipelines including phase 1 DB-020, that will have initial human efficacy data expected in 2021. This newly acquired funding will help to advance their pipeline DB-OTO, which is in the preclinical stage and have been shown to restore hearing in children with congenital deafness due to a dysfunctional otoferlin gene, and Decibel expects to initiate clinical testing in 2022.
‘At Decibel, we are dedicated to restoring hearing and balance with precision therapeutics designed to deliver the right genetic medicine specifically to the right cells in the ear. We are grateful to this group of outstanding investors for choosing to partner with us to advance this vision.”- Laurence Reid, Ph.D., CEO, Decibel therapeutics.
Novo Nordisk acquires Emisphere Technologies for $1.35 billion
On 6th November 2020, Denmark-based pharmaceutical company Novo Nordisk acquired Emisphere Technologies for the oral drug delivery platform Eligen® which facilitates the enhanced oral absorption of molecules without altering their chemical form, biological integrity, or pharmacological properties. The merger deal is worth $1.35 billion. These two companies have been partnering since 2007. Utilizing an existing license agreement Novo Nordisk is currently utilizing Rybelsus®, Emisphere’s carrier technology for the oral formulation of a GLP-1 receptor agonist semaglutide. Novo Nordisk is also spending $450 million to acquire related Eligen® SNAC royalty stream obligations owed to MHR Fund Management LLC (MHR). The total buyout deal rounds up to $1.8 billion.
“After a thorough analysis of strategic alternatives, the Emisphere Board and the Special Committee unanimously determined that a combination with Novo Nordisk is the best way to maximize value for our stockholders,” - Timothy G. Rothwell, Chairman, Emisphere.
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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