This week's life sciences industry news includes firms making progress on experimental treatments for brain cancer and depression, a discovery that could help HIV patients, a potentially big deal in Big Pharma, and more.
Here is the roundup:
Imvax
The Philadelphia immunotherapy company said it dosed its first patient in a Phase 2 clinical trial for IGV-001, its experimental therapy for a type of brain cancer known as glioblastoma. The treatment is designed to stimulate a patient’s immune system against the entire antigen signature of their tumor.
The study intends to enroll a total of up to 93 patients to evaluate the safety and effectiveness of IGV-001 in newly diagnosed glioblastoma patients.
John P. Furey, CEO of Imvax, called the company's progression into this Phase 2b clinical study a "significant milestone" for both Imvax and the many patients and families facing a glioblastoma diagnosis.
“For too long, there have been few advancements in the treatment of glioblastoma," Furey said.
The primary endpoint of the trial is progression-free survival, and key secondary endpoints include overall survival and safety.
GSK
The London-based pharmaceutical giant, which has operations in Philadelphia and Montgomery County, entered into an exclusive license and commercialization agreement with Scynexis (NASDAQ: SCYX) for Brexafemme.
Brexafemme is an antifungal approved by the Food and Drug Administration to treat vulvovaginal candidiasis, a vaginal yeast infection, and to reduce incidences of recurrent infections.
The deal gives GSK (NYSE: GSK) rights to commercialize Brexafemme while continuing to develop ibrexafungerp, the drug's active ingredient, which is in late-stage testing as a potential treatment of invasive candidiasis, a life-threatening fungal infection.
Under the financial terms of the agreement, GSK will pay Scynexis $90 million up front plus potential milestone-based payments totaling $503 million.
Wistar Institute
Researchers at the independent Philadelphia biomedical research center have identified a potentially promising compound for people living with HIV.
The natural plant-based compound called hopeaphenol targets HIV reservoirs that persist in people living with HIV despite the presence of anti-HIV therapy, according to Wistar.
“This is important because anti-HIV therapy can stop the symptoms, but it doesn't eliminate the potential of the underlying HIV reservoir from re-emerging," said Ian Tietjen, a research assistant professor with Wistar’s Vaccine & Immunotherapy Center and the lead author of the scientific paper Wistar published about the compound. "The virus is still there and still a little bit active — kind of rumbling and turning on — and the immune system is stressed about that.”
According to Tietjen, the expression of persistent HIV can stress immune cells even in the presence of anti-HIV therapy, which has been linked to inflammation and higher risk of cancers, metabolic disease, heart disease, and other conditions associated with advanced aging in persons living with HIV.
“We think that something like hopeaphenol could prevent the HIV reservoir from reactivating — reducing stress on the immune system and potentially decreasing these age-related conditions,” he said.
NRx Pharmaceuticals
The Radnor biopharmaceutical company said an independent data safety monitoring board has recommended that enrollment in the trial for NRx's lead new drug candidate, NRX-101, continue as planned.
NRX-101 is being developed as a potential treatment for severe bipolar depression and subacute suicidal ideation or behavior.
The monitoring board reviewed the safety and efficacy findings of the first 50 enrolled participants in the company's Phase 2/3 clinical trial of NRX-101, a proprietary fixed-dose combination of D-cycloserine and lurasidone. According to NRx, the board found no futility signal and no safety issues at this stage of the trial.
NRx (NASDAQ: NRXP) expects to report data form the trial during the fourth quarter of 2023.
Quick Hits
Cabaletta Bio (NASDAQ: CABA), a Philadelphia cell therapy developer, received FDA approval to begin human clinical testing of CABA-201, the company's experimental treatment for systemic lupus erythematosus. The autoimmune disease, which affects an estimated 160,000 to 320,000 patients in the United States, causes a person's immune system to attack its own tissues, causing widespread inflammation and tissue damage in affected organs. … Wilmington-based Incyte (NASDAQ: INCY) received approval for Pemazyre from the Japanese Ministry of Health, Labour and Welfare as a treatment of myeloid/lymphoid neoplasms. Myeloid/lymphoid neoplasms are a rare, aggressive group of cancers characterized by an over-production of myeloid cells, or bone tissue. The condition, which afflicts fewer than 100 patients worldwide, has the tendency to rapidly progress to an acute myeloid leukemia. … Baudax Bio (NASDAQ: BXRX) of Malvern announced positive results from its second preplanned interim analysis of its Phase 2 trial of BX1000. BX1000 is a neuromuscular blockade under development for patients undergoing elective surgery. The company expects to announce study results in late April or early May. … Ocugen (NASDAQ: OCGN) received FDA approval to enroll pediatric patients in its ongoing OCU400 Phase 1/2 trial. The gene therapy candidate is under development as a potential treatment for inherited retinal diseases such as retinitis pigmentosa and leber congenital amaurosis. Ocugen, based in Malvern, plans to initiate a Phase 3 trial for OCU400 near the end of 2023.
