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MedNess: bite-size biopharma and medtech news

3rd March, 2021
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MedNess This Week
HIGHLIGHTS
Onco-I-Analyse
On 24th February, Kura oncology announced that the US FDA has granted breakthrough therapy designation to Tipifarnib (selective inhibitor of farnesyl transferase) as a treatment option for recurrent or metastatic HRAS mutant (allele frequency ≥ 20%) head and neck squamous cell carcinoma (HNSCC) following progression on platinum-based chemotherapy. The designation is based on preliminary data from the Phase 2 RUN-HN study presented during ASCO in May 2020.
Background: Head and neck squamous cell carcinoma is the 7th leading cancer in the world with a 5-year survival rate of ~40% in patients with distant metastasis. Patients progressing on frontline therapy usually have an ORR of 6-16%, mPFS and mOS of of 2-3 and 5-8 months, respectively. Nearly 4-8% patients with HNSCC have HRAS mutation.
Details: RUN-HN is a non-randomized study evaluating tipifarnib in patients with HRAS-mutant (with a variant allele frequency (VAF) > 20%) tumors including recurrent or metastatic HNSCC in 63 patients. HNSCC patients with a median of 2 prior therapies had received platinum-based chemotherapy (90.5%), immunotherapy (61.8%), and cetuximab (52.4%).
The trial demonstrated an ORR of 50%, mPFS of 5.9 months (vs. 2.8 months on last line of therapy), mOS of 15.4 months and mDOR of 14.7 months in 18 response-evaluable patients.
In December 2019, tipifarnib was granted Fast Track Designation for the treatment of patients with HRAS mutant HNSCC.
Implications: Tifiparnib is currently being evaluated in a pivotal Phase 2 AIM-HN study investigating the asset in HRAS mutation positive HNSCC and the impact of HRAS on response to 1stL systemic therapies. The study is expected to support accelerated approval for tipifarnib.
Collated by : Shilpa Rawal, PhD
Drug Approvals
FDA approved Libtayo® (cemiplimab-rwlc) in 1L EGFR/ALK/ROS-1 WT, PD-L1-high NSCLC patients
"The approval of Libtayo to treat first-line advanced non-small cell lung cancer with high PD-L1 expression means physicians and patients have a potent new treatment option against this deadly disease," said Naiyer Rizvi, M.D., Price Family Professor of Medicine, Director of Thoracic Oncology and Co-director of Cancer Immunotherapy at Columbia University Irving Medical Center, as well as a steering committee member of the trial. "Notably, Libtayo was approved based on a pivotal trial where most chemotherapy patients crossed over to Libtayo following disease progression, and that allowed for frequently underrepresented patients who had pretreated and clinically stable brain metastases, or who had locally advanced disease and were not candidates for definitive chemoradiation. This gives doctors important new data when considering Libtayo for the varied patients and situations they treat in daily clinical practice."
Regulatory News
Voluntary withdrawal of Imfinzi indication in advanced bladder cancer in the US
Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: “The science of immunotherapy has moved swiftly over the past few years, bringing new options to patients at an unprecedented pace. While the withdrawal in previously treated metastatic bladder cancer is disappointing, we respect the principles FDA set out when the accelerated approval pathway was founded and remain committed to bringing new and innovative options to patients. In the last three years, Imfinzi has become an important standard of care in multiple lung cancer settings, an area of considerable focus for AstraZeneca.”
FDA Fast Track Designation for GEN-1 in Advanced Ovarian Cancer
“Fast Track designation is an important step in developing GEN-1 for advanced ovarian cancer. Presuming the encouraging data we are generating in early clinical studies continues, this designation supports an expedited path to market,” said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. “Fast Track allows for more frequent communication with the FDA to discuss development plans and clinical trial design. In addition, should criteria be met, Fast Track-designated drugs are eligible for rolling review, a process whereby the drug’s sponsor can separately submit sections of its New Drug Application to the FDA. They also are eligible for accelerated approval and priority review, under which drugs for serious conditions fulfilling an unmet medical need can be approved based on a surrogate endpoint. We are optimistic that GEN-1 represents a game-changer for women with advanced ovarian cancer who have limited treatment options.”
Click here for more Regulatory News
Trial Results
Ide-cel produces remissions in almost 75% of RRMM patients who relapsed after several previous treatments in the Ph 2 KarMMa trial; data published in NEJM
“Despite numerous advances in the treatment of multiple myeloma, relapses are common. Patients whose disease continues to worsen after receiving standard therapy have relatively few treatment options that provide high response rates,” said Nikhil Munshi, MD, of Dana-Farber Cancer Institute, who led the trial. “The results of this trial represent a true turning point in the treatment of this disease. In my 30 years of treating myeloma, I have not seen any other therapy as effective in this group of patients.”
Clinical Update Provided on Ph 1/2 OVATION 2 Study with GEN-1 in Advanced Ovarian Cancer Including Encouraging Interim Resection Scores
“As the goal for surgical debulking is to eliminate microscopic disease, more ovarian cancer patients require neoadjuvant chemotherapy. However, little progress has been made in adding additional efficacious immunotherapy agents to standard neoadjuvant chemotherapy,” said Premal H. Thaker, M.D., MSc., Professor in Gynecologic Oncology and Director of Gynecologic Oncology Clinical Research at Washington University School of Medicine in St. Louis and lead Principal Investigator for the OVATION 2 Study. “The results seen to date in the OVATION 2 Study are exciting and impactful for ovarian cancer patients.”
Click here for more Trial Results
Trial/Program Status
Clinical program for Berubicin on track to start enrolling rGBM patients in March 2021
"I am very pleased with our progress and the team's execution towards the start of our program. We have made significant advancements and are now finalizing clinical site selection and preparing to begin patient screening, which we expect to commence next month," commented John Climaco, CEO of CNS Pharmaceuticals. "Berubicin's promising results demonstrated in the Phase 1 clinical trial build on sixty years of clinical experience with anthracyclines.  Berubicin is an entirely novel molecule that represents an opportunity to recognize the powerful benefits of this tried-and-true class of drugs for neuro-oncology in general and in the fight against GBM in particular. We are deeply committed to driving this program forward as expeditiously as possible with the prime focus on our mission to improve patient outcomes for GBM."
IDMC recommends Advancing the OVAL Ph 3 Registration Enabling Study of VB-111 in Ovarian Cancer
"This review continues the trend of encouraging reviews that have taken place since the clinical trial began," said Prof. Dror Harats, Chief Executive Officer of VBL Therapeutics. "The trial continues to enroll on track in the US, Europe and Israel. We look forward to the next DSMC review during the third quarter of 2021, followed by completion of enrollment at the end of 2021 or in early 2022.”
Click here for more Trial Statuses
Collated by : Richa Tewari, PhD
Genes and Therapy
Launch of first-in-human clinical trials to assess BDNF gene therapy for Alzheimer’s disease
Researchers at the University of California San Diego School of Medicine have launched the Phase I clinical trial for gene therapy for Alzheimer’s Disease to improve memory. The therapy will deliver the BDNF (brain-derived neurotrophic factor) gene packed inside a harmless adeno-associated virus (AAV2) at targeted regions of the brain that usually gets affected by Alzheimer’s, namely, the entorhinal cortex and hippocampus. BDNF is critical to maintain existing neurons and support the growth of new neurons and synapses. The protein is particularly important in regions susceptible to neurodegeneration. This three-year-long trial will recruit 12 participants diagnosed with either Alzheimer’s Disease or Mild Cognitive Impairment, with another 12 persons serving as comparative controls over that period. The trial is presently recruiting patients.
Jaguar Gene Therapy launches to accelerate breakthrough in gene therapies for severe genetic diseases
Jaguar Gene Therapy launched on 25th February 2021 with a mission to accelerate the development, manufacturing and commercialization of novel gene therapy treatments for patients suffering from severe genetic diseases. The company is founded by former management team from AveXis and Series A funded by Deerfield Management. Their current pipeline utilizes the proven and well-characterized AAV9 vector for gene therapy.  Their initial pre-clinical pipeline targets four diseases including galactosemia, genetic causes of autism spectrum disorder, Type 1 diabetes and Bardet-Biedl syndrome.
Click here for more on Genes and Therapy
Medness Business
Onco-News
Compugen Expands Clinical Collaboration Agreement with BMS with Ph 1b Combination Study of COM701 with Opdivo®
"We are excited to further expand our clinical program evaluating COM701, our first-in-class anti PVRIG inhibitor," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "While our triple checkpoint blockade study of COM701 combined with Bristol Myers Squibb's PD-1 and TIGIT inhibitors currently advancing in the clinic offers the ultimate test of our science-driven hypothesis, translational research at Compugen suggests that certain patients may not require a triple therapy combination. With the enrollment in the dose escalation arm of COM701 in combination with Opdivo® completed and preliminary signs of antitumor activity previously disclosed, we are ready to continue our evaluation of this dual combination and move to the cohort expansion phase of the study. Testing COM701 in three settings – as a monotherapy, dual combination, and triple combination therapy – may provide additional insights on the contribution of components as well as the opportunity to broaden COM701 treatment options to address patients' needs. We are proud to be moving quickly to initiate this biomarker and data-informed study in indications we believe are most likely to respond to dual PVRIG and PD-1 blockade, enhancing our leadership position in the DNAM-1 axis space."

Day One Expands Clinical-Stage Oncology Pipeline; Announces Global License Agreement with Merck to Develop and Commercialize MEK Inhibitor Pimasertib
“Day One is purpose-built to accelerate innovative targeted therapies designed to help both children and adults with cancer,” said Jeremy Bender, Ph.D., chief executive officer of Day One. “This license agreement with Merck KGaA, Darmstadt, Germany, exemplifies our core strategy to identify investigational potential treatment options such as pimasertib and leverage our expertise to rapidly advance them in patients who we believe will benefit the most. We are excited that a leading pharmaceutical company like Merck KGaA, Darmstadt, Germany recognizes the importance of our mission and look forward to the advancement of pimasertib in combination with our pan-RAF kinase inhibitor, DAY101.”
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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