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MedNess: bite-size biopharma and medtech news
2nd June, 2021
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MedNess This Week
HIGHLIGHTS
Drug Approvals
FDA Approves LUMAKRAS™ (Sotorasib), The First And Only Targeted Treatment For Patients With KRAS G12C-Mutated Locally Advanced or mNSCLC
"The FDA approval of LUMAKRAS is a breakthrough moment for patients with KRAS G12C-mutated non-small cell lung cancer because there is now a targeted therapy for this common, but previously elusive, mutation," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "KRAS has challenged cancer researchers for more than 40 years with many deeming it as 'undruggable.' The LUMAKRAS development program was a race against cancer for Amgen's scientists and clinical trial investigators who together have now successfully delivered this new medicine to patients in less than three years—from first patient dosed to U.S. regulatory approval."
FDA approves TRUSELTIQ™ (infigratinib) for patients with cholangiocarcinoma
“This is an important milestone for patients diagnosed with FGFR2-fusion-driven cholangiocarcinoma who have recurred after first-line therapy and are in need of targeted options for further treatment,” said Susan Moran, M.D., M.S.C.E., Chief Medical Officer for QED. “Based on the efficacy seen to date, our team believes infigratinib possesses promise for a range of FGFR-driven conditions, including other cancers. We will continue to evaluate its safety and efficacy in these areas of unmet need.”
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Regulatory News
US FDA Grants BCMA CAR-T Cilta-cel Priority Review for the Treatment for R/R Multiple Myeloma
“Cilta-cel has shown great promise in the treatment of patients with heavily pretreated multiple myeloma according to study findings reported to date. Today’s priority review designation marks another significant milestone for this cell therapy,” said Ying Huang, PhD, CEO and CFO of Legend Biotech. “We look forward to our continued collaborative efforts with Janssen and in working with the FDA to bring this transformative therapy to patients who are in need of new treatment options.”
First patient dosed in EG-70 Ph 1/2 trial for the Treatment of Non-Muscle Invasive Bladder Cancer
"Initiating our first clinical study of EG-70 is an important milestone for enGene, validating the potential of our platform to develop novel therapeutics towards clinically meaningful outcomes," said Jason Hanson, Chief Executive Officer at enGene. "This is the first time a non-viral gene therapy directly activating both innate and adaptive immunity is being evaluated as an anti-tumor agent in humans, and we are excited about the outcome of this trial towards positively impacting the lives of patients battling difficult-to-treat cancers."
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Trial Results
Primary Endpoint Met in Ph 3 Study of the Anti-PD-L1 Antibody Sugemalimab in Stage III NSCLC
“Stage III NSCLC represents a heterogeneous group of patients with a wide range of therapeutic outcomes. Around the world, both sequential and concurrent chemotherapy are commonly used treatment approaches for this stage of disease,” said Vincent Miller, MD, physician-in-chief at EQRx. “These encouraging results from both the Stage III and Stage IV studies suggest sugemalimab is a promising potential treatment option in a broad range of patient populations.”
Camidanlumab Tesirine Ph 1 Results Published in The Lancet Haematology
“There is a significant unmet medical need for novel therapies that improve outcomes in patients with relapsed or refractory Hodgkin lymphoma,” said Mehdi Hamadani, MD, Professor of Internal Medicine at the Medical College of Wisconsin, Division of Hematology & Oncology and lead author of The Lancet Haematology paper. “This patient population is often heavily pretreated, as was the case in this published study in which patients experienced a median of five previous systemic therapies. The Phase 1 study demonstrates encouraging potential for Cami to provide a new treatment option for patients with relapsed or refractory Hodgkin lymphoma.”
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Trial/Program Status
First Patient Dosed in Ph 3 Clinical Trial of TAVT-45 (abiraterone acetate) Granules for Oral Suspension, a Novel Formulation of Abiraterone Acetate, for the Treatment of Metastatic Prostate Cancer
“We are excited to be the first clinical trial site to dose a patient in this trial” said Dr. Robert Given, a urological oncologist at Urology of Virginia and a study investigator. “The current standard of care treatment requires patients to ingest multiple large tablets, and under strict fasting conditions due to the risk of increased and highly variable abiraterone exposures with food. In contrast, TAVT-45 Granules has the potential to provide the more than 3.2 million prostate cancer patients in the United States, specifically those with dysphagia, with an alternative option that would reduce pill burden.”
Enrollment opens at participating hospitals in Europe for a Ph 3 maintenance study evaluating selinexor in patients with endometrial cancer after combination chemotherapy
"While selinexor has been most extensively studied in patients with hematologic malignancies, there is increasing evidence that selinexor may also play an important role in the treatment of a variety of solid tumors, including patients with endometrial cancer," said Sharon Shacham, PhD, MBA, Chief Scientific Officer of Karyopharm. "As there are currently no approved drugs in the U.S. or Europe to treat patients with endometrial cancer in the maintenance setting following chemotherapy, the SIENDO study has the potential to help meaningfully advance the treatment paradigm for patients in need of new options for advanced or recurrent endometrial cancer."
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Collated by : Richa Tewari, PhD
MedNess Reviews

Scientists Develop Inhalable Nanobody for Treatment of SARS-CoV2 Infection in Hamsters
Scientists at the University of Pittsburgh have shown that low doses of tiny antibody fragment known as a “nanobody” when administered directly through the nose by inhalation, could prevent and treat severe COVID-19 in hamsters.

The treatment, dubbed PiN-21, was created after the team identified over 8,000 nanobodies against the coronavirus. They picked the most potent candidate in that showed promising results in-vitro and engineered it further to better interact with the coronavirus to create PiN-21.

The treatment could offer an affordable, needle-free option for the treatment early COVID infections and it may also help high-risk patients.

Nanobodies, are gaining attention as alternatives to monoclonal antibody therapies. Nanobodies are fragments of conventional monoclonal antibodies. Originally identified in llamas and other camelid, they show potential in addressing the deficiencies of monoclonal antibody therapies due to their small size and lower molecular weight.

In their study, the team tested PiN-21in Syrian hamsters. They administered PiN-21 into the nasal cavities after animals were infected by the virus through the trachea or intranasally and found that the animals that received the treatment did not experience any significant weight loss and cleared the virus in the lungs after 10 days when compared to animals that received a placebo.

The tiny size of the nanobody raised the possibility that they could be efficiently delivered to the lung by aerosolization. To test this, the researchers also aerosolized the nanobodies using a nebulizer and dosed the hamsters. They found that when PiN-21 was aerosolized, even a ultralow dose of the treatment revered weight loss in animals infected with the virus when compared to control animals and reduced the number of infectious virus particles in the lung tissue by six orders of magnitude, or a million-fold. The scientists also observed a substantial reduction of viral levels in nasal washes and throat swabs.

Existing monoclonal antibody treatments such as Regeneron’s REGEN-COV cocktail offer potent treatment options for COVID-19 however, they are required in high doses to be effective against the disease, and the high costs and manufacturing challenges also limit their potential for broad clinical application.

Nanobodies treatments such as PiN-21, hold potential for effective, low-dose and needle free options for the treatment of SARS-CoV2 infection.
MedNess Business
Onco-News
QIAGEN Partners With Mirati Therapeutics Inc. to Develop KRAS-G12C Companion Diagnostic for NSCLC
“We are pleased Mirati recognizes the success of QIAGEN’s therascreen platform and continues to partner with us to develop a tissue-based companion diagnostic to identify patients who may benefit from adagrasib. QIAGEN’s experience and expertise in developing diagnostic solutions for Precision Medicine are well-suited to evaluate patients with non-small cell lung cancer,” said Jean-Pascal Viola, Senior Vice President and Head of QIAGEN’s Molecular Diagnostics Business Area. “Our collaboration with Mirati is a demonstration of QIAGEN’s capabilities as a preferred partner of pharmaceutical and biotech companies for the creation of companion diagnostics.”
Xilio Therapeutics Announces Clinical Trial Collaboration with Merck on Anti-CTLA-4 Monoclonal Antibody Program
“We believe tumor-selective immuno-oncology has significant potential to provide meaningful treatments to patients with a number of different cancers. The clinical benefit of targeting CTLA-4 as a treatment for cancer is well-established. However, treatment with the combination of an antiCTLA-4 mAb and PD-1 checkpoint inhibitor has been associated with challenging toxicities, preventing patients from receiving effective doses of the anti-CTLA-4 antibody,” said Marty Huber, M.D., chief medical officer of Xilio Therapeutics. “XTX101 is designed to be tumor-selective and based on data observed in preclinical studies, we believe it could be an ideal CTLA-4-targeting candidate to combine with checkpoint inhibitors like KEYTRUDA. We are pleased to partner with Merck to study this combination in an effort to improve therapeutic options for people with cancer.”
Click here for more on mergers, acquisition and business news
Collated by : Richa Tewari, PhD
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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Content Editors: Richa Tewari , Esha Sehanobish , Rinki Saha ,  Shilpa Rawal, PhD ,  Debarati Banik  , Divyaanka Iyer , Arundithi Ananthanarayanan and Abhinav Dey 
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