Target $6
Monday, May 2, 2022
Vertex: FDA halts Phase 1/2 diabetes study
-Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today provided updates on its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemia. Two patients in Part A received VX-880 at half the target dose. The first patient dosed in Part A of the study achieved insulin independence at Day 270, with a HbA1c of 5.2%. The second patient dosed in Part A has shown robust increases in fasting and stimulated C-peptide, and reductions in exogenous insulin requirements through Day 150. Taken together, the data from the first two patients in Part A established proof-of-concept for VX-880. Per the study protocol, the Independent Data Monitoring Committee reviewed the totality of the safety and efficacy data from the first two patients dosed in Part A of the study and recommended advancement to Part B, where patients receive the full target dose of VX-880. The first patient to receive the full target dose has achieved the Day 29 follow-up milestone.
Across the program, VX-880 has been generally well tolerated to date. There have been no serious adverse events (SAEs) considered related to VX-880. The majority of adverse events (AEs) were mild or moderate in all patients treated to date. The safety profile was generally consistent with the immunosuppressive regimen used in the study and the perioperative period.
The company also announced the VX-880 Phase 1/2 study has been placed on clinical hold in the U.S. by the Food and Drug Administration (FDA) due to a determination that there is insufficient information to support dose escalation with the product.
"We are surprised by the clinical hold placed on the study. The results from the first two patients treated with half the target dose establish proof-of-concept by demonstrating that VX-880 can restore glucose-regulated insulin production and improve glycemic control. Indeed, achievement of insulin independence by the first patient is a landmark milestone. Further, the totality of the safety and efficacy data for all three patients dosed to date gives us high confidence in our benefit-risk assessment of VX-880 and its potentially transformative profile," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "We are committed to working with the FDA to understand and address their questions, so that the trial can resume at U.S. sites as soon as possible."
https://finance.yahoo.com/news/vertex-provides-updates-phase-1-123000988.html
Cue Health Asks FDA for Full Clearance of Its Molecular COVID-19 Test
Cue Health ("Cue") (Nasdaq: HLTH) today became the first company to submit to the U.S. Food and Drug Administration (FDA) for full clearance of its molecular COVID-19 test for at-home and point-of-care use. FDA clearance would provide the public with a molecular COVID-19 test that has been fully reviewed by the FDA for safety and effectiveness.
A prior submission to the FDA for Emergency Use Authorization of Cue's COVID-19 at-home, over-the-counter test showed 98.9% accuracy. An independent study by Mayo Clinic showed Cue's COVID-19 test to be in 97.8% overall concordance with central lab PCR testing.
"This FDA submission marks a major milestone for the company and begins to define a new space of molecular testing in the home and at the point-of-care. We hope this will be the first of many submissions for Cue's molecular testing as we look to address a range of diseases and conditions and make healthcare more responsive, convenient, and effective," said Ayub Khattak, CEO and co-founder of Cue Health.
https://finance.yahoo.com/news/cue-health-makes-novo-submission-130000144.html
Therapeutic Solutions Starts Phase 3 Trial for Treating COVID-19 Lung Damage
Therapeutic Solutions International announced today the launching of a double-blind, randomized, placebo controlled, multi-center, multi-nation, clinical trial of 128 patients with COVID-19 associated lung failure.
The study will be comprised of two groups, JadiCell™ treatment group and control group. The primary endpoint of the study is comparing the proportion of patients alive and free of respiratory failure at Day 60 after treatment with JadiCells as compared to placebo.
The Company has licensed the issued patent covering composition of matter for JadiCell and FDA Right of Reference1,2, has identified and filed patents on novel mechanisms of action of JadiCell related to lung preservation and regeneration3,4,5, and acquired the FDA cleared IND and associated data package, which was the basis for Phase III clearance6, and has contracted Biorasi, a global, full-service CRO, to launch and run the clinical trial.
"Cell therapy is one of the most promising and demanding forms of medical intervention," said Chris O’Brien, CEO of Biorasi. "In contrast to traditional medicines, products such as JadiCell are living therapeutics, which require a very detail-oriented approach to their administration as well as patient follow-up. We are honored to partner with TSOI for this landmark clinical trial."
In previous studies the Company has demonstrated the superior activity of JadiCell to other types of stem cells including bone marrow, adipose, cord blood, and placenta. Furthermore, the JadiCell was shown to be 100% effective in saving the lives of COVID-19 patients under the age of 85 in a double-blind, randomized, placebo controlled clinical trial with patients in the ICU on a ventilator. In patients over the age of 85 the survival rate was 91%7.
Stealth misses primary endpoint in Phase 2 trial
Study did not meet primary endpoints of mean change in low luminance visual acuity (LLVA) and geographic atrophy (GA) progression
Categorical improvement in LLVA, with >15% of elamipretide-treated patients gaining 2+ lines of vision at Week 48
Elamipretide demonstrated enhanced ellipsoid zone preservation through reduction of progressive attenuation, an important indicator of photoreceptor loss.
Company to host conference call and webcast today at 8:30 a.m. ET
Conference Call Information
Stealth will host a conference call and webcast today at 8:30 am ET to discuss the ReCLAIM-2 trial. The call can be accessed by dialing 1-877-407-0989 (toll free) or 1-201-389-0921 (international) and referencing conference ID 13729736. A live audio webcast of the event can be accessed by visiting the Investors & News section of Stealth's Investor website, https://investor.stealthbt.com/. A replay of the webcast will be archived on Stealth's website for 30 days following the event.
https://finance.yahoo.com/news/stealth-biotherapeutics-announces-data-reclaim-113000750.html
Gilead, Dragonfly to Develop Natural Killer Cell Engagers in Oncology and Inflammation
Gilead Sciences, Inc. (Nasdaq: GILD) and Dragonfly Therapeutics today announced a collaboration designed to advance a number of Dragonfly's novel natural killer (NK) cell engager-based immunotherapies for oncology and inflammation indications. NK cell engagers represent a novel mechanism with the potential to address a broad range of cancers, including potential for activity in checkpoint resistant and refractory tumors, as well as other disease areas such as inflammation. Under the agreement, Gilead will receive an exclusive, worldwide license from Dragonfly for the 5T4-targeting investigational immunotherapy program, DF7001. The agreement also grants Gilead options, after the completion of certain preclinical activities, to license exclusive, worldwide rights to develop and commercialize additional NK cell engager programs using the Dragonfly Tri-specific NK Engager (TriNKET™) platform. TriNKETs are activators of the innate and adaptive immune systems, recruiting NK and cytotoxic T cells into the tumor microenvironment.
https://finance.yahoo.com/news/gilead-dragonfly-announce-strategic-research-123000112.html
Vaxxinity Fast Tracked for Treatment of Alzheimer’s Disease
Vaxxinity, Inc. (Nasdaq: VAXX), a company pioneering the development of a new class of immunotherapeutic vaccines, today announced that UB-311, an anti-amyloid beta immunotherapeutic vaccine, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of Alzheimer’s disease.
“We are excited that the FDA has granted UB-311 Fast Track Designation, as it recognizes the evidence demonstrating the potential for UB-311 to address a serious unmet medical need for patients with Alzheimer’s disease,” said Mei Mei Hu, Chief Executive Officer of Vaxxinity. “We are on an encouraging clinical path for UB-311 and look forward to collaborating with the FDA and other global regulatory agencies to bring UB-311 expeditiously to the global market. Because our vaccine approach allows for more convenient administration and broad access, UB-311 is positioned to potentially lead a paradigm shift in the treatment, and even prevention, of Alzheimer’s.”
https://finance.yahoo.com/news/vaxxinity-receives-fda-fast-track-120000998.html
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