Target $162
Monday, February 22, 2021
FDA: BrainStorm's ALS Cell Therapy Lacks Substantial Data For Submission
The FDA's initial review concluded that current data from BrainStorm Cell Therapeutics Inc's (NASDAQ: BCLI) NurOwn Phase 3 trial in amyotrophic lateral sclerosis (ALS) does not sufficiently provide the threshold of substantial evidence to support the marketing application.
Also, the FDA advised that this recommendation does not preclude Brainstorm from proceeding with a marketing application submission.
"Brainstorm will first consult with principal investigators, ALS experts, expert statisticians, regulatory advisors, and ALS advocacy groups to assess the benefit/risk of a BLA submission before making a final decision," said Chaim Lebovits, CEO.
NurOwn, autologous MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells that have been expanded and differentiated ex vivo.
The cells can deliver immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.
Celsion Fast Tracked in Advanced Ovarian Cancer
Celsion Corporation (NASDAQ: CLSN), a clinical stage development company focused on DNA based immunotherapy and next generation vaccines, today announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for GEN-1, its DNA-mediated interleukin-12 (IL-12) immunotherapy currently in Phase II development for the treatment of advanced ovarian cancer. GEN-1 was designed using TheraPlas, Celsion's proprietary, synthetic, non-viral nanoparticle delivery system platform.
Fast Track designation is intended to facilitate the development and expedite the regulatory review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, a Fast Track Drug must show some advantage over available therapy, including:
- Showing superior effectiveness, effect on serious outcomes or improved effect on serious outcomes
- Avoiding serious side effects of an available therapy
- Decreasing a clinical significant toxicity of an available therapy that is common and causes discontinuation of treatment
“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.”
GEN-1 is the subject of Celsion’s Phase II OVATION 2 Study, which combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).
As Celsion has previously announced, it has shared with the FDA data from the Phase I portion of the Phase I/II OVATION 2 Study that showed successful tumor resections, with seven out of eight patients (88%) in the GEN-1 treatment arm having a complete tumor resection (R0), which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed. The NACT-only treatment arm had an R0 resection rate of 50%.
UBS Starts Ortho Clinical Diagnostics (OCDX) at Buy
UBS analyst John Sourbeer initiates coverage on Ortho Clinical Diagnostics (NASDAQ: OCDX) with a Buy rating and a price target of $23.00.
The analyst comments "We are initiating coverage of OCDX with a Buy rating and $23 PT. OCDX is a pure play IVD company with an attractive Razor / Razor blade model focusing on Clinical Labs and Transfusion Medicine. OCDX has historically grown revenues LSD and we model revenues accelerating to MSD driven by instrument placements, customer upgrades and exposure to the fast growing China market. We see self-help levers such as margin expansion from operating efficiencies and leverage reduction in addition to revenue acceleration providing DD EPS growth. We see valuation upside if revenues accelerate to the MSD range and leverage approaches the company's 3-3.5x target range."
Adamis OKd for Human Studies of Tempol for Treatment of COVID-19
Adamis Pharmaceuticals Corporation (Nasdaq: ADMP) ("Company") announced today that the U.S. Food and Drug Administration (“FDA”) has completed the safety review of the Company’s Investigational New Drug (“IND”) application and has concluded that Adamis may proceed with the proposed clinical investigation of Tempol for the treatment of COVID-19. The clearance to proceed follows the submission of an IND application to FDA and a Pre-IND meeting.
The goal of the study titled, “A Phase 2/3, Adaptive, Randomized, Double-Blind, Placebo-Controlled Study to Examine the Effects of Tempol (MBM-02) on Preventing COVID-19 Related Hospitalization in Subjects with COVID-19 Infection,” is to examine the safety and activity of Tempol in COVID-19 patients early in the infection. In addition to safety, the study will examine markers of inflammation and the rate of hospitalization for patients taking Tempol versus placebo early in COVID-19 infection. More details of the protocol can be found here or by searching Clinicaltrials.gov.
“As the principal investigator of this study, I am excited to initiate this clinical trial to evaluate the role of Tempol to prevent serious complications and hospitalization in COVID-19 patients. We are in need of additional therapeutic options for COVID-19, and this novel antioxidant approach deserves a thorough investigation,” noted Shyam Kottilil, MBBS, PhD, Professor of Medicine and Director of the Division of Clinical Care and Research at the Institute of Human Virology at the University of Maryland School of Medicine.
Tempol has demonstrated both potent anti-inflammatory, anticoagulant, and antioxidant activity. Both inflammatory cytokines and reactive oxygen species (ROS) from cells of the immune system called macrophages and neutrophils damage the lung in Acute Respiratory Distress Syndrome (ARDS). In animal models, Tempol has been shown to decrease proinflammatory cytokines (cytokine storm), and through its potent antioxidant activity has been shown to decrease the harmful effects of ROS. In addition, Tempol has been shown to decrease platelet aggregation, a problem observed in many COVID-19 patients.
Incyte Gets Priority Review for Jakafi in Chronic Graft-Versus-Host Disease
Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for ruxolitinib (Jakafi®) for treatment of steroid-refractory chronic graft-versus-host disease (GVHD) in adult and pediatric patients 12 years and older.
The sNDA submission is based on results from the Phase 3, randomized REACH3 study comparing ruxolitinib with best available therapy (BAT) in patients with steroid-refractory chronic GVHD. In the REACH3 study, which was recently presented at the 62nd American Society of Hematology (ASH) Annual Meeting & Exposition, patients treated with ruxolitinib experienced a significantly greater overall response rate (ORR) compared to BAT at Week 24, the primary endpoint (49.7% vs. 25.6%; p<0.0001). For the key secondary endpoints, ruxolitinib was associated with a longer median failure-free survival (FFS) than BAT at Week 24 (not reached vs. 5.7 months; hazard ratio (HR), 0.370; p<0.0001), and greater symptom improvement per the modified Lee Symptom Scale (mLSS) at Week 24 (24.2% vs. 11.0%; odds ratio (OR), 2.62; p=0.0011). The best ORR for patients receiving ruxolitinib was 76.4%. No new safety signals were observed, and adverse events were consistent with the known safety profile of ruxolitinib.
“Chronic GVHD is a life-threatening complication following stem cell transplant that burdens a vulnerable patient population, which today has limited treatment options,” said Peter Langmuir, M.D., Group Vice President, Oncology Targeted Therapies, Incyte. “The acceptance of this sNDA represents an important milestone for Incyte as we continue our work towards helping more people living with GVHD, particularly for those who do not respond to steroids. We look forward to working closely with the FDA to bring this innovative therapy to patients and to providing continued support to the GVHD community in the United States.”
GVHD is a condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) in which the donated cells initiate an immune response and attack the transplant recipient’s organs, leading to significant morbidity and mortality. There are two major forms of GVHD: acute, which generally occurs within 100 days of transplant, and chronic, which generally occurs after 100 days of transplant1. Both forms can affect multiple organ systems, including the skin, gastrointestinal (digestive) tract and liver.
The FDA grants Priority Review to medicines that may offer a major advance in treatment where none currently exists. This designation shortens the review period to six months compared to 10 months for Standard Review. The Prescription Drug User Fee Act (PDUFA) target action date for Jakafi in steroid-refractory chronic GVHD is June 22, 2021.
The sNDA is also being reviewed as part of the Project Orbis program, an initiative of the U.S. FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology drugs among international regulatory agencies. Participating countries for this application include Canada, Australia, Switzerland, Brazil and the United Kingdom.
In 2019, Jakafi was approved by the U.S. Food and Drug Administration for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older2.
Arcturus Acquires Exclusive License to mRNA Manufacturing Technology from Alexion
Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a leading clinical-stage messenger RNA medicines company focused on the development of infectious disease vaccines and significant opportunities within liver and respiratory rare diseases, today announced that the Company has acquired an exclusive license from Alexion Pharmaceuticals to certain patent-pending inventions relating to nucleic acid purification technologies.
Since the Company’s inception in 2013, Arcturus has been building proprietary expertise related to the manufacture of RNA through technological innovation. The licensed inventions enable larger scale and faster production of mRNA. The technology supports the Company’s efforts to optimize the highly efficient manufacture of mRNA therapeutic candidates with an increased purity profile. This acquisition extends the substantial intellectual property portfolio already held by Arcturus for the manufacture of high purity pharmaceutical quality mRNA therapeutic candidates for a variety of diseases.
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